Regulatory tracker: Merck's Keytruda picks up 2 new approvals in Europe

By Angus Liu, Kevin Dunleavy, Eric Sagonowsky, Fraiser Kansteiner Dec 19, 2023 8:40am

Fierce Pharma is reintroducing the Pharma Regulatory Tracker. Here, we're recording the regulatory progress of in-market products, including expansions into key geographies and new indications. Some of these updates may not make the bar for standalone stories, but we think they are still worth mentioning.

UPDATED: Tuesday, December 19 at 8:40 a.m.

The number of approved indications for Merck's star cancer medicine Keytruda has grown to 26 in Europe.

Most recently, the European Commission approved the drug—in two chemotherapy combinations—for separate first-line uses in gastrointestinal cancers.

The first approval covers the use of Keytruda, plus fluoropyrimidine- and platinum-containing chemotherapy, for the first-line treatment of locally advanced unresectable or metastatic HER2-negative gastric or gastroesophageal junction adenocarcinoma in certain adults whose tumors express PD-L1.

Additionally, the EC signed off on Keytruda's use in combination with gemcitabine and cisplatin for the first-line treatment of adults with locally advanced unresectable or metastatic biliary tract carcinoma.

Europe's Committee for Medicinal Products for Human Use (CHMP) previously endorsed these uses based on data from Merck's KEYNOTE-859 and KEYNOTE-966 studies, respectively.
Also in Europe, Biogen said that the CHMP granted a positive opinion for Skyclarys to treat Friedreich’s ataxia in people 16 and older.

If the EC signs off on the drug's approval as expected, Skyclarys would be the first Friedreich’s ataxia medicine in Europe.

In a 48-week study called MOXIe Part 2, patients who received the drug had less physical impairment, as measured by the modified Friedreich Ataxia Rating Scale, than those who received placebo.

Biogen picked up Skyclarys, a potential blockbuster, when it acquired Reata Pharmaceuticals earlier this year.
Lastly, Ultragenyx's treatment for an ultrarare, inherited form of high cholesterol won an expanded approval from the European Commission.

The EC endorsed Evkeeza as an adjunct to diet and other medicine to treat children ages 5 to 11 with homozygous familial hypercholesterolemia. The approval builds on the medicine's first nod in patients 12 and older back in 2021.

UPDATED: Friday, December 15 at 10:15 a.m.

Following approvals in the U.K. and the U.S., Vertex's CRISPR-based gene-editing therapy Casgevy has picked up a positive opinion from the European Medicines Agency (EMA).

The European agency recommends an approval for Casgevy for transfusion‑dependent beta thalassemia and severe sickle cell disease in patients 12 years of age and older who are eligible for a stem cell transplant but who don't have a suitable donor.

The EMA reviewed Casgevy's application under its priority medicines scheme, and the therapy is recommended for a conditional marketing authorization. Once Casgevy is approved by the European Commission, Vertex is on the hook to submit the final results from its pivotal trials by August 2026, as well as results from an ongoing long-term follow-up study, among other data. Patients who got Casgevy will be followed up for 15 years.
Despite GSK's appeal, the EMA's Committee for Medicinal Products for Human Use decided not to renew a conditional approval for the company's multiple myeloma drug Blenrep.

The EU regulator based its decision on findings from the DREAMM-3 trial, which showed that Blenrep couldn't outdo Bristol Myers Squibb's Pomalyst and dexamethasone at slowing disease progression or death in previously treated patients. GSK last year pulled Blenrep from the U.S. market because of the same flop.

The EMA will now send its opinion to the European Commission for a final decision. Once the decision is formally adopted as expected in Feburary 2024, GSK will work with the EU member states to initiate withdrawal of Blenrep, a GSK spokesperson told Fierce Pharma.

The regulatory setback doesn't affect GSK's plan for Blenrep in other combinations, the spokesperon said. The company recently announced positive findings from the DREAMM-7 trial, showing Blenrep extended progression-free survival compared with Johnson & Johnson’s Darzalex in their respective combinations with Takeda’s Velcade and the steroid dexamethasone in multiple myeloma patients who had tried at least one prior line of treatment.
In addition to Casgevy, the EMA also recommended approvals for Pfizer's Velsipity in moderately to severely active ulcerative colitis, Biogen/Reata's Skyclarys for Friedreich’s ataxia and Takeda's HyQvia as a maintenance therapy for chronic inflammatory demyelinating polyneuropathy.

The agency also expanded its recommendation for Sanofi's Fexinidazole Winthrop to treat Trypanosoma brucei (T.b.) rhodesiense sleeping sickness. Years ago, the drug was recommended for the more common T.b. gambiense form of sleeping sickness found in West and Central Africa.

UPDATED: Thursday, December 14 at 8:55 a.m.

After a recent meeting with European regulators, Apellis said it has been "informed of a negative trend vote" on its filing for pegcetacoplan to treat geographic atrophy (GA) secondary to age-related macular degeneration (AMD).

In a Thursday update, Apellis said it expects Europe's Committee for Medicinal Products for Human Use to "adopt a negative opinion" on the application.

If that happens, Apellis said it will appeal and seek a re-examination of the filing. That process could take 4 to 5 months, Mizuho analysts said in a Thursday note.

“We are very disappointed in this outcome and saddened for the millions of GA patients in Europe who have no treatment for this leading cause of blindness,” Apellis' chief development officer, Jeffrey Eisele, Ph.D., said in a statement.

Apellis is leveraging its phase 3 Oaks and Derby studies for the filing. The drug picked up its FDA approval in GA back in February.
In the U.S., Michigan-based drugmaker Esperion has received several label updates for its cholesterol-busting drugs Nexletol and Nexlizet.

Thanks to FDA nods in 2020 and 2021, the drugs have been approved to treat patients with heterozygous familial hypercholesterolemia or atherosclerotic cardiovascular disease who require additional lowering of LDL-C.

In a recent update, the FDA added primary hyperlipidemia as a qualifier for treatment in the approved patient populations. Further, the agency removed a requirement that patients try a "maximally tolerated" statin dose before starting on the Esperion drugs.

Esperion is still aiming for a label addition detailing the drugs' cardiovascular risk reduction, with a decision on that filing expected by the end of March. The company also expects European approval to come in the first half of 2024.

UPDATED: Monday, December 11 at 9:55 a.m.

GSK's PD-1 cancer drug Jemperli has picked up a European approval to treat adult patients with mismatch repair deficient (dMMR)/microsatellite instability-high (MSI-H) primary advanced or recurrent endometrial cancer who are candidates for systemic therapy. The approval covers the use of Jemperli in combination with carboplatin-paclitaxel in this group of patients.

With the nod, Jemperli becomes the first frontline immuno-oncology treatment for this patient population in Europe, GSK said in a Monday release.

Besides the frontline approval, the European Commission converted a prior conditional second-line approval in dMMR/MSI-H recurrent or advanced endometrial cancer into a full nod.

Results from the phase 3 Ruby study enabled the new approval. In the study, investigators noted a 72% reduction in the risk of disease progression or death for the Jemperli regimen compared with chemotherapy alone.

"With this approval, we can expand the number of patients who can potentially benefit from treatment with Jemperli in Europe, including patients who are earlier in their journey," GSK's global oncology R&D head, Hesham Abdullah, said in a statement.
Also in Europe, Astellas' Veoza has gained regulatory approval to treat moderate to severe vasomotor symptoms (VMS) associated with menopause.

The approval follows an October recommendation from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) in favor of a marketing authorization. More than 3,000 participants took part in phase 3 trials enabling the regulatory nod.

In the U.S., the drug won its FDA nod back in May. It's marketed as Veozah in the States.

UPDATED: Friday, December 8 at 10:00 a.m.

A few months after picking up an FDA approval, Pfizer's Elrexfio has gained a regulatory nod from the European Commission.

Specifically, the EC approved the drug to treat patients with relapsed and refractory multiple myeloma who have received at least three prior therapies, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody.

Pfizer's drug is an off-the-shelf, BCMA-directed bispecific antibody that's administered subcutaneously. Elrexfio gained its FDA approval in August.

The European nod advances Pfizer's clash with Johnson & Johnson and its rival bispecific antibody Tecvayli. J&J's drug won its EU nod this past summer and its FDA accelerated approval in October 2022.
As the FDA is scrutinizing Bristol Myers Squibb's CAR-T drug Abecma in earlier lines of treatment. The BCMA-targeted therapy has won a label expansion in Japan.

This week, regulators in the country approved the cell therapy to treat patients with relapsed or refractory multiple myeloma who have received at least two prior therapies.

Results from BMS' KarMMa-3 enabled this label expansion. The study tested the CAR-T drug in patients who've tried between two and four prior treatments, delivering a statistically significant increase in progression-free survival compared with a standard treatment.

Abecma won its original approval in Japan back in January 2022 to treat patients after three prior lines of therapy.

UPDATED: Wednesday, December 6 at 10:05 a.m.

After European drug regulators issued a negative opinion on renewing Translarna's conditional marketing authorization, PTC Therapeutics has responded as part of a regulatory "re-examination procedure" that's slated to last until late January.

Afterward, the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) will issue a final opinion on whether the Duchenne muscular dystrophy therapy should remain on the market in Europe. And the European Commission will have around 2 months to accept or reject that decision, PTC explained in a Tuesday release.

In the U.S., meanwhile, the company is working with the FDA on identifying a path to a drug resubmission. Recently, the FDA told the company that its data do not support an application filing. The company anticipates having a meeting with the regulator to go over its updated data in the first quarter of 2024.

Translarna is licensed in the U.K. and Europe, but it remains an investigational drug in the U.S.
Phathom Pharmaceuticals announced that the FDA accepted its label expansion application for Voquezna to treat heartburn associated with non-erosive gastroesophageal reflux disease (GERD).

The FDA has set a target action date of July 19, 2024, for this filing.

Last month, Phathom's Voquezna, a potassium-competitive acid blocker, picked up FDA approvals to treat Helicobacter pylori infection and erosive GERD.

Before those approvals, Phathom ran into a regulatory setback when trace levels of a cancer-causing agent were discovered in the drug.

UPDATED: Tuesday, December 5 at 11:00 a.m.

Bristol Myers Squibb's application for Opdivo and cisplatin-based chemotherapy as a first-line treatment for metastatic urothelial carcinoma has scored an FDA priority review.

The agency has set a target decision date of April 5, 2024, for the filing, BMS said in a release.

Bristol's CheckMate-901 trial supported the application. If approved, the combo would be the first immunotherapy-chemotherapy regimen for this patient group, according to BMS.

Bladder cancer is the 10th most common cancer type worldwide, according to Bristol Myers. Urothelial carcinoma accounts for the vast majority of bladder cancer cases.
Meanwhile, ImmunoGen's effort to convert Elahere's accelerated approval into a full FDA nod has also gained a priority review.

Like Bristol's filing, the agency set April 5 of next year as its decision date for the ImmunoGen filing.

Elahere is currently approved under the FDA's accelerated pathway to treat patients with folate receptor alpha-positive, platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer who have received one to three prior systemic treatment regimens.

The company's supplemental FDA application leverages results from the phase 3 MIRASOL trial. Earlier this year, the company touted the result as a "home run."

More recently, AbbVie agreed to purchase ImmunoGen for around $10 billion. Elsewhere, Elahere is also under review in Europe and China, ImmunoGen said.

UPDATED: Monday, December 4 at 10:45 a.m.

Shortly after gaining an FDA approval, UCB's Zilbrysq has scored a thumbs up to treat generalized Myasthenia Gravis (gMG) in Europe.

The European Commission has approved the drug to treat gMG in adult patients who are anti-acetylcholine receptor (AChR) antibody-positive, UCB said in a Monday release. This comes about six weeks after the medicine won an approval in the U.S.

The company's therapy is the first once-daily subcutaneous, targeted C5 complement inhibitor for this disease, UCB said.

Zilbrysq is UCB's second drug for gMG after the FDA approved Rystiggo in June.

The approval is supported by data from the company's phase 3 RAISE study, which showed the med outperformed placebo in MG-specific efficacy metrics.
Last week, the FDA warned about the potential for a "rare but serious" reaction to certain antiseizure drugs.

Specifically, the agency said levetiracetam and clobazam can cause Drug Reaction with Eosinophilia and Systemic Symptoms (DRESS), which is life-threatening if not treated quickly.

DRESS can start as a rash but progress quickly and cause damage to organs, the FDA said.

The agency is adding warning language about the reaction to the drugs' labels, and it's recommending users seek "immediate medical attention" for any unusual symptoms they start to experience.

UPDATED: Friday, December 1 at 11:00 a.m.

After the European Medicines Agency (EMA) in July kicked off a review of GLP-1 medicines over reports of patients experiencing suicidal thoughts, the regulator is continuing its probe and asking companies for more information.

Drugs from Novo Nordisk, Eli Lilly, Sanofi and AstraZeneca are involved in the review, Reuters reports.

In a Friday update, EMA said that its Pharmacovigilance Risk Assessment Committee is requesting "further clarifications" from the companies marketing these medicines.

"While at this point no conclusion can be drawn on a causal association, there are several issues that still need to be clarified," the EMA said Friday.
The FDA has accepted Merck, Seagen and Astellas' application for the combination of Keytruda and Padcev for the treatment of adult patients with first-line, locally advanced or metastatic urothelial carcinoma.

With the filing acceptance, the FDA has offered priority review and set a target action date of May 9, 2024.

In the high-profile Phase 3 EV-302 trial, the combo showed a statistically significant and clinically meaningful improvement in overall survival and progression-free survival compared to chemotherapy.
Chinese authorities granted emergency use authorization to a domestic mRNA COVID vaccine against Omicron XBB.1.5, a securities filing (Chinese) to the Shenzhen Stock Exchange shows.

Coded RQ3033, The shot was developed by Walvax, Fudan University and Shanghai RNACure Biopharma.

Meanwhile, CSPC Pharma, whose COVID shot Duentai in March became the first mRNA vaccine cleared in China, got emergency use clearance for its bivalent vaccine targeting XBB.1.5 and BQ.1, accoring to a securities filing (PDF) to the stock exchange of Hong Kong.
Following damning side effect reports tied to Apellis' geographic atrophy injection Syfovre, the FDA has officially added the issue of retinal vasculitis with or without occlusion to the drug's label, Apellis said Friday. The issue is now reflected in the “Warnings and Precautions” and “Adverse Reactions - Postmarketing Experience” sections of C3 inhibitor's label.

The rate of retinal vasculitis is estimated at 0.01% per injection, with more than 120,000 injections estimated to have been administered as of Nov. 18, Apellis said.

UPDATED: Wednesday, November 29 at 9:25 a.m.

Novavax's updated COVID-19 vaccine, Nuvaxovid XBB.1.5, has been granted an Emergency Use Listing from the World Health Organization, the company said in a Tuesday release.

The regulatory pathway assists the WHO's 194 member states in assessing vaccine safety and efficacy and enables faster national approvals, the company explained.

While Novavax's COVID vaccine never gained the level of uptake as its rivals, the company touts its shot's supply-chain requirements as one advantage of the product.

Novavax's vaccine can be stored at 2 to 8 degrees Celsius and has a 12-month shelf life.

"Rural or hard-to-reach areas can benefit from our vaccine's ease of transport and storage profile," Novavax CEO John Jacobs said in a statement. "As part of a diversified vaccine portfolio, our vaccine can play an important role in helping to protect people around the globe against the latest variants."
Arcutis said the FDA has accepted its supplemental new drug application for roflumilast cream to treat atopic dermatitis in adults and children down to age 6.

The company's new application seeks approval for a 0.15% dose of the cream. The FDA has set a July 7, 2024, decision deadline for this application.

At a higher 0.3% dose, roflumilast cream is approved as Zoryve for the topical treatment of plaque psoriasis, including intertriginous areas, in patients 6 years of age and older.
After England's National Institute for Health and Care Excellence’s (NICE) recommended against Kyowa Kirin's Crysvita to treat X-linked hypophosphataemia, the company said it remains committed to working with stakeholders to make the drug available to patients.

In an interim decision, NICE did not recommend the drug to treat adults who have a confirmed diagnosis of XLH. The decision could lead to a disparity in treatment between patients in England, Wales and Scotland, the company noted, because Scottish authorities blessed the drug earlier this year.

The decision in Scotland shows that the drug "can be clinically beneficial and cost effective for this group of patients," Kwoya Kirin said in a release. Ten European countries already reimburse use of the medicine.

UPDATED: Monday, November 27 at 8:20 a.m.

AstraZeneca has struck a commercial deal with officials at NHS England to enable access to Kanuma for patients with Wolman disease. With the deal, the drug is now recommended for use by the National Institute for Health and Care Excellence (NICE).

Also known as sebelipase alfa, Kanuma is an enzyme replacement therapy that becomes the first treatment available on NHS England for Wolman disease, a rapidly-progressing and life-threatening genetic condition that causes damage to multiple organs. The disease is estimated to occur in about 1 in 350,000 births.

With the Monday recommendation, the drug will be "fast-tracked" to any eligible patients, NHS England said in a release.

AstraZeneca acquired Kanuma as part of its 2021 buyout of Alexion Pharmaceuticals.
AbbVie and Genmab's Epkinly has picked up an FDA Breakthrough Therapy Designation for patients with relapsed or refractory follicular lymphoma (FL) who have tried at least two other therapies.

The drug previously won an FDA accelerated approval to treat diffuse large B-cell lymphoma (DLBCL) after at least two lines of systemic therapy.

Aside from the FDA breakthrough designation in the FL use, the European Medicines Agency validated a Type II application for the drug in the same indication.

Results from the Phase 1/2 EPCORE NHL-1 clinical trial supported the recent regulatory advancements.

UPDATED: Monday, November 20 at 9:55 a.m.

Johnson & Johnson has submitted an FDA application for its Rybrevant—in combination with chemotherapy—to treat certain patients with locally advanced or metastatic non-small cell lung cancer (NSCLC).

Specifically, the company aims to win a label expansion to treat patients with epidermal growth factor receptor exon 19 deletions or L858R substitution after disease progression on or after osimertinib. Osimertinib is the generic name for AstraZeneca's Tagrisso.

J&J is leveraging data from the phase 3 MARIPOSA-2 study for the new filing.

This potential label expansion is one step for the drug toward reaching J&J's blockbuster revenue expectations. Rybrevant won its original FDA approval in 2021 to treat NSCLC patients whose tumors bear exon 20 mutations and who have progressed on or after platinum-based chemotherapy.
Sanofi's Dupixent has picked up an approval in China to treat asthma in people 12 and older, China Daily reports.

Asthma is Dupixent's sixth approved indication in China and the drug's first respiratory use in the country, according to the publication.

Sanofi has gained 10 approvals for new drugs or label expansions this year, Sanofi's Greater China president Wayne Shi said in a statement.

UPDATED: Friday, November 17 at 8:40 a.m.

The European Commission has signed off on Almirall's Ebglyss to treat patients 12 and older with moderate to severe atopic dermatitis. Patients must weigh at least 40 kilograms (88 pounds) to be eligible for the new treatment.

Almirall plans to start its European rollout in Germany, with other launches following throughout Europe next year.

Almirall nabbed European rights to the drug, also known as lebrikizumab, under a licensing deal with Eli Lilly. In the U.S., the drug recently suffered an FDA rejection due to shortcomings at a third-party manufacturer.
Also in Europe, BeiGene's Brukinsa scored regulatory approval to treat patients with relapsed or refractory follicular lymphoma (FL) who've tried two prior therapies. The approval covers Brukinsa in combination with Roche's Gazyvaro.

With the approval, Brukinsa becomes the first BTK inhibitor to win approval for FL in the European Union.

The approval was based on results from the phase 2 ROSEWOOD trial, which tested the Brukinsa and Gazyvaro combo versus Gazyvaro alone. Investigators recorded an overall response rate of 69% for the combo, versus 45.8% for the Gazyvaro monotherapy arm.
Following an FDA approval in June for argenx's Vyvgart Hytrulo, the European Commission has followed suit.

Thursday, argenx announced European approval of subcutanous Vyvgart for adults with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor antibody positive.

After the nod, argenx says it will work with local health authorities in EU member countries to enable patient access to the new option.
Avistone Biotechnology has picked up the first global approval for vebreltinib.

Friday, the company said China's National Medical Products Administration approved the medicine to treat patients with MET exon 14 skipping non-small cell lung cancer.

The approval is based on the phase 2 KUNPENG study, which showed the drug was associated with an objective response rate of 75% in that patient group. The median progression-free survival result was 14.1 months in those patients.

Investigators recently presented the results at the European Society for Medical Oncology's meeting in Madrid.

UPDATED: Friday, November 10 at 11:35 a.m.

Two days after the FDA approved Eli Lilly’s tirzepatide to treat obesity under the brand name Zepbound, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended the drug in the same indication.

The drug is currently approved to treat patients with Type 2 diabetes under the brand moniker Mounjaro but has been increasingly used off-label as an obesity therapy.
The CHMP has also adopted a positive opinion for Bayer's high-dose Eylea, for which Regeneron got the FDA's go-ahead in August.

The recommendation opens 8-mg Eylea for formal European Commission approval for age-related macular degeneration and diabetic macular edema. Compared with the original version, the high-dose formulation allows for extended treatment intervals of up to every four months.
During the week, the CHMP also backed GSK's momelotinib for the treatment of myelofibrosis. The drug's EU brand name will be Omjjara.

Similar to the drug's FDA label, European regulators are supporting Omjjara's use in both JAK-naive patients and those who have got Incyte and Novartis' Jakavi.

In two other CHMP reommendations, Blueprint Medicines' Ayvakit is poised to expand into indolent systemic mastocytosis, and Pfizer's Talzenna is on track to a metastatic castration-resistant prostate cancer indication in adults for whom chemotherapy is not clinically indicated.
Meanwhile, Novartis has recently withdrawn its EMA application for Vijoice for the genetic condition of PIK3CA-related overgrowth spectrum, which is marked by abnormal growth or tumours affecting several tissues.

The FDA approved Vijoice in April 2022. But in a letter to the EMA, Novartis said that it needed more time to obtain additional data to support Vijoice's application.
Bristol Myers Squibb has scored a priority review from the FDA for its application to expand the label of its CAR-T Breyanzi.

The application is to treat patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who have received treatment with a BTK inhibitor and BCL-2 inhibitor. The FDA has established a PDUFA date of March 14 of next year.

The application is based on results from a phase 1/2 study, which is the first to show clinical benefit with a CAR-T cell therapy in patients with relapsed or refractory CLL after disease progression following treatment with a BTKi and BCL2i.
The FDA has agreed to expand the label of Pacira BioScience’s Exparel, a non-narcotic, non-opioid treatment that reduces pain locally after surgery.

Exparel now can be used as an adductor canal block for anesthesia for surgeries of the knee, medial lower leg and ankle. A sciatic nerve block in the popliteal fossa can be used for anesthesia with surgeries of the foot, ankle and Achilles tendon.

UPDATED: Monday, November 6 at 11:15 a.m.

The FDA is reviewing Amgen’s application for a biosimilar to Regeneron and Bayer’s macular degeneration blockbuster Eylea, Generics Bulletin reported Monday. The news comes after Amgen previously posted positive results from its phase 3 biosimilar comparison trial in neovascular age-related macular degeneration, or wet AMD.

Eylea is headed for the patent cliff on May 17, 2024, though some analysts figure copycats won’t enter the playing field until 2025, the news outlet said.

Several other drugmakers, like Formycon and Biocon, are also awaiting FDA decisions on biosimilar Eylea applications. In the case of Biocon—which recently bought Viatris’ biosimilars business—the company’s copycat prospect is embroiled in a lawsuit with Regeneron over alleged patent infringement.

Over the first nine months of the year, Eylea generated $6.98 billion in global sales, Regeneron reported last week. Regeneron shares Eylea with Germany’s Bayer, which holds exclusive marketing rights to the drug outside of the United States.
The FDA is putting healthcare providers on alert following a potential misadministration trend around Moderna’s COVID-19 vaccine in kids.

The agency says it’s learned that some healthcare workers may not recognize that a single vial of Moderna’s shot for children 6 months through 11 years of age contains “notably more” of the vaccine than the approved 0.25mL dose. Practitioners could be withdrawing the entire contents of the vial to administer to an individual patient, the FDA warned.
The United Kingdom’s Medicines and Healthcare Products Regulatory Agency (MHRA) gave a thumbs up to Mirati Therapeutics’ KRAS inhibitor Krazati in adults with advanced non-small cell lung cancer (NSCLC) whose disease has progressed after prior chemotherapy or treatment with a PD-1/PD-L1 immunotherapy.

Patients’ disease must also bear a KRAS G12C mutation to qualify for Mirati’s oral therapeutic.

Lung cancer is one of the most common cancers globally, accounting for 2.21 new cases and some 1.8 million deaths worldwide in 2020, according to Mirati. KRAS G12C is the most common mutation in NSCLC.

The U.K. approval leveraged results from KRYSTAL-1, an open-label phase 1/2 multiple-expansion cohort trial looking at Krazati alone and in combination with other drugs in patients with KRAS-mutated advanced solid tumors.

Krazati snared U.S. approval back in December, becoming the second therapy to take aim at KRAS after Amgen’s Lumakras.

It hasn’t all been smooth sailing for Mirati’s first commercial product. In July, the European Medicines Agency (EMA) rebuffed the company’s conditional marketing authorization application for Krazati—a decision Mirati claimed it disagreed with.
Roche’s bispecific follicular lymphoma (FL) drug Lunsumio has crossed the regulatory threshold in Korea, thanks to a green light from the country’s Ministry of Food and Drug Safety, Korea Biomedical Review reports. With the nod, Lunsumio becomes the first therapeutic to receive approval through the regulator’s fast track program.

Korea’s drug regulator cleared the drug using data from the phase 2 expansion study GO29781, which enrolled patients with recurrent or refractory follicular lymphoma who’d tried at least two prior systemic therapies.

Lunsumio snagged its third-line FL approval in the U.S. in December, thanks to data showing the antibody shrank tumors in 80% of patients with heavily pretreated disease. Sixty percent of those patients also hit complete remission.

Because the drug also won an accelerated nod in the U.S., Lunsumio is subject to a confirmatory phase 3 trial.

UPDATED: Thursday, November 2 at 9:05 a.m.

Phathom Pharmaceuticals' Voquezna has won a label expansion from the FDA to treat adults with all grades of erosive esophagitis, which is also known as gastroesophageal reflux disease.

The approval marks the first "major innovation" in the field in more than 30 years, according to the company's press release.

The drug won its original approval last May to treat H. pylori infections. But Phathom later had to put the launch on hold to get a handle on impurities that turned up in certain commercial batches.

Now, Phathom is gearing up for a launch next month. Also, with the FDA's approval, the company gets its hands on $175 million based on the terms of a prior financing deal.

That financing will help the company fund the launch, Phathom said.
Pfizer's alopecia areata drug Litfulo has secured an approval in the U.K.

Wednesday, authorities with the Medicines and Healthcare products Regulatory Agency (MHRA) signed off on the drug to treat severe alopecia areata in patients 12 and older.

Pfizer's Litfulo, also known as ritlecitinib, is an enzyme inhibitor that won its U.S. FDA approval back in June.

UPDATED: Monday, October 30 at 10:55 a.m.

As concerns mount in the U.S. and abroad over potential side effects from popular GLP-1 drugs such as Ozempic, Wegovy and Trulicity, Novo Nordisk, Eli Lilly and others have eluded certain safety concerns in Europe.

The European Medicines Agency’s safety committee (PRAC), after looking at available evidence on glucagon-like peptide-1 (GLP-1) drugs, said the class of diabetes and obesity drugs does not appear to be linked to the risk of thyroid cancer.

The EMA issued its bill of clean health after signaling a ramp-up of its GLP-1 scrutiny this summer. Stateside, many GLP-1s, including Novo’s Ozempic and Wegovy, as well as Lilly’s Trulicity and its newer Type 2 diabetes offering Mounjaro, carry boxed warnings about the potential risk of users developing thyroid cancer.
The European Medicines Agency signed off on Bristol Myers Squibb’s Opdivo application, teeing up a review of the immunotherapy plus cisplatin-based chemo for previously untreated unresectable or metastatic bladder cancer.

The win comes courtesy of phase 3 data from BMS’ CheckMate-901 trial, which the company recently touted at the European Society for Medical Oncology (ESMO) annual meeting. In the late-stage study, the Opdivo cocktail lowered the risk of death from bladder cancer by 22% and extended the median time patients lived by 2.8 months to 21.7 months.

CheckMate-901 also enrolled patients who can’t take cisplatin, but that portion of the study hasn’t read out yet.

BMS is pursuing a similar bladder cancer nod for Opdivo in the United States, where the medicine could go on to compete with Merck KGaA’s Bavencio—approved as a first-line maintenance therapy in patients who haven’t had diseased progression following initial platinum-containing chemotherapy. That said, Opdivo’s could be facing an even bigger rival in Astellas, Seagen and Merck & Co.’s combination of PD-1 inhibitor Keytruda and the antibody-drug conjugate (ADC) Padcev.

UPDATED: Friday, October 27 at 10:00 a.m.

Servier's IDH1 inhibitor Tibsovo has picked up its fifth indication worldwide.

Earlier this week, the cancer specialist said its drug has gained FDA approval to treat patients with isocitrate dehydrogenase 1 (IDH1)-mutated relapsed or refractory myelodysplastic syndromes.

Tibsovo is now the first and only FDA-approved targeted therapy for this patient group, Servier said.

Aside from this use, Tibsovo is approved by the FDA to treat IDH1-mutated acute myeloid leukemia in certain situations. The agency has also endorsed the med for previously treated IDH1-mutated cholangiocarcinoma.

Elsewhere, Tibsovo boasts approvals in Europe, Australia and China.

UPDATED: Wednesday, October 25 at 9:35 a.m.

Belgium's health minister said officials there are looking at a temporary ban of Novo Nordisk's Ozempic as a weight-loss treatment, Reuters reports.

The country has already instructed doctors to reserve the medicine for patients with type 2 diabetes, but that measure did not go far enough, Franck Vandenbroucke told the local broadcaster RTBF.

A potential ban could last weeks or months, according to Reuters. The discussions come amid a shortage of the popular drug.
Akebia, working to regain momentum after a high-profile rejection last year, said the FDA has accepted its new drug application for vadudastat.

The company is seeking an approval for vadudastat to treat anemia due to chronic kidney disease in adult patients on dialysis. The FDA is set to decide on the filing by March 27, 2024.

Outside the U.S., vadadustat is approved in 34 countries, including Japan, where the drug carries the Vafseo moniker. The drug was approved by the European Commission in late April.

Akebia suffered a surprise FDA rejection for its medicine in March 2022, prompting the company to file a formal complaint in October 2022.
Samsung Bioepis and Biogen's biosimilar to Genentech's popular eye drug Lucentis has won a coveted FDA interchangeability designation.

The designation should give the biosimilar, Byooviz, a boost as it fights for market share in a competitive field.

Biogen and Samsung Bioepis launched their biosimilar in the U.S. last June.

UPDATED: Monday, October 23 at 3:20 p.m.

AstraZeneca and Daiichi Sankyo's Enhertu has received a new approval from the European Commission to treat certain adults with advanced non-small cell lung cancer.

Specifically, the drug is now approved as a monotherapy for patients whose tumors have an activating HER2 mutation and who require systemic therapy following platinum-based chemotherapy with or without immunotherapy.

Results from the partners' DESTINY-Lung02 study informed the decision. Previously, the Committee for Medicinal Products for Human Use recommended the drug be approved in this use.

Enhertu becomes the first HER2-directed therapy for this patient population to win approval in Europe, the companies said.
Celltrion has picked up an FDA nod for Zymfentra, the first subcutaneous biosimilar to Johnson & Johnson's immunology drug Remicade.

The Celltrion drug is approved as a maintenance therapy for moderately to severely active ulcerative colitis or Crohn’s disease after intravenous treatment with an infliximab product.

The subcutaneous drug holds patent protection until 2037 for its dosage form and 2040 for its route of administration, Celltrion said.

Celltrion's intravenous infliximab biosimilar, Remsima, is already approved in around 110 countries.
Ipsen is navigating some regulatory challenges for its rare disease drug Bylvay in Europe.

Most recently, the company said the European Medicines Agency’s Committee for Orphan Medicinal Products confirmed a prior opinion recommending against the drug's orphan designation in Alagille syndrome.

Orphan designations carry "strong influence" over reimbursement mechanisms and patient access in some European countries, Ipsen said.

With the latest decision, the company plans to submit its drug to the EMA with a new proposed brand name for treating Alagille syndrome by the end of the year.

In July, a separate EMA committee recommended the drug for approval in Alagille syndrome.

UPDATED: Thursday, October 19 at 2:57 p.m.

Takeda's antibody-drug conjugate (ADC) Adcetris has picked up an approval from the European Commission to treat adults with previously untreated CD30+ stage III Hodgkin lymphoma.

The approval covers Adcetris' use in this setting in combination with doxorubicin, vinblastine and dacarbazine (AVD).

Before the nod, the CD30-directed ADC had already won six different approvals in Europe, Takeda said in a release.

The approval leveraged phase 3 data showing that the Adcetris and AVD combo delivered a progression-free survival benefit over doxorubicin, bleomycin, vinblastine, and dacarbazine (ABVD). The Adcetris regimen also improved overall survival.

Last month, the European Medicines Agency's Committee for Medicinal Products for Human Use threw its weight behind the drug's approval in this use.
Orasis Pharmaceuticals, a Florida-based ophthalmic pharmaceutical company, has secured an FDA approval for its Qlosi to treat presbyopia, or the age-related inability to clearly see nearby objects.

In two phase 3 trials that enrolled more than 600 participants, Qlosi eye drops "consistently demonstrated efficacy, safety, and tolerability," the company said in a release.

The drops can be used daily or as needed, up to two times per day.

Qlosi's vision benefits can start as soon as 20 minutes after administration and can last up to 8 hours, trials showed.

UPDATED: Monday, October 16 at 8:15 a.m.

Novavax has encountered a setback in its attempt to win market authorization for its updated COVID-19 vaccine in Europe, The Financial Times reports.

The European Medicines Agency was set to decide on approving the vaccine by Friday, but it instead sent the filing back to Novavax with a request for more information.

A Novavax spokesperson told FT that the company is responding "expeditiously." Two weeks ago, Novavax won FDA authorization for its updated vaccine in people 12 and older.
AstraZeneca has picked up an FDA priority review for the Tagrisso and chemotherapy combo in locally advanced or metastatic EGFR-mutated non-small cell lung cancer (NSCLC).

With the speedy review, the regulator is expected to decide on the combo therapy during the first quarter of 2024, AstraZeneca said.

AZ's approval application is based on data from AstraZeneca's Flaura2 study, which showed that the combo cut the risk of disease progression or death by 38% compared to Tagrisso alone. Tagrisso is the first-line global standard of care in this disease, AstraZeneca said.
The European Commission has approved Merck's Keytruda as a monotherapy for the adjuvant treatment of adults with NSCLC who are at high risk of recurrence following complete resection and platinum-based chemotherapy.

The decision follows a recommendation for approval from Europe's drug regulator last month.

This indication represents Keytruda's fifth lung cancer approval in Europe, Merck said.

UPDATED: Friday, October 13 at 10:35 a.m.

In Europe, the Committee for Medicinal Products for Human Use (CHMP) recommended several new medicines for approval this week.

The drugs that have gained CHMP's backing are Santhera's Agamree for Duchenne muscular dystrophy, Pfizer's multiple myeloma therapy Elrexfio, Guerbet's MRI imaging agent Elucirem, Immedica's rare-disease drug Loargys, plus Melinta's antifungal Rezzayo and Astellas' Veoza to treat vasomotor symptoms associated with menopause.

The recommendations now head to the European Commission for a final sign-off.
In addition to the recommendations for new drug approvals, CHMP endorsed label expansions for 8 drugs. Merck's Keytruda, BeiGene's Brukinsa, GSK's Jemperli and AstraZeneca's Imfinzi were among the cancer meds to get line-expansion recommendations.
Meanwhile, two companies are coming back to the CHMP to request re-examinations of recent reviews.

Last month, the CHMP voted against renewing marketing authorizations for PTC Therapeutics' DMD drug Translarna and GSK's multiple myeloma therapy Blenrep. Both companies have asked for re-examinations of the CHMP's assessment, according to the group's Friday update.

UPDATED: Tuesday, October 10 at 10:30 a.m.

Novartis scored an FDA approval for an intravenous (IV) version of its IL-17A antagonist Cosentyx, adding a new option to the previously only subcutaneous treatment.

The new version was approved for all of Cosentyx’s existing indications, which includes psoriatic arthritis, ankylosing spondylitis and non-radiographic axial spondyloarthritis.

Now, the med is the first IV treatment to target IL-17A and the only non-TNF infusion on the market for those conditions, Novartis said. The IV option allows healthcare providers “choice and flexibility” to tailor to different patients needs, the company added in its statement.
Gilead Sciences’s Kite saw a win in Scotland as the country’s Scottish Medicines Consortium accepted Kite’s CAR-T Tecartus on its National Health Service (NHS).

The therapy was accepted to treat relapsed or refractory B-cell precursor acute lymphoblastic leukaemia, an aggressive and rare blood cancer.

In what Scotland’s Medicines Consortium’s chair Dr. Scott Muir called a “record making month” for the regulator, it also accepted several other cancer treatments including AstraZeneca’s Lynparza, BeiGene’s Brukinsa and Bayer’s Stivarga and Nubeqa onto its NHS.
Supernus is trying again with its apomorphine infusion device for continuous treatment of motor fluctuations in Parkinson’s disease.

After an October 2022 complete response letter, the company resubmitted its application under the belief that it has since addressed the FDA’s questions and concerns.

The complete response letter didn’t ask for additional safety and efficacy studies, but required “additional information and analysis” and a manufacturing inspection, Supernus said in a statement.

UPDATED: Friday, October 6 at 9:50 a.m.

Pfizer's Abrilada, the company's biosimilar version of AbbVie's Humira, has won an interchangeability designation from the FDA.

The tag means that the drug can be substituted at the pharmacy counter by a pharmacist. Abrilada is only the second Humira biosim to gain the designation, following Boehringer Ingelheim's Cytelzo.

Meanwhile, Pfizer still hasn't launched its biosimilar. Eight other Humira biosims are now on the market, according to The Center for Biosimilars.

Pfizer says it plans to launch the product "later this year at two price points."
Arcutis' Zoryve has won a label expansion approval treat plaque psoriasis in children ages 6 to 11.

The drug is a once-daily, steroid-free cream that won its original FDA approval last year to treat plaque psoriasis in patients 12 and older.

The label expansion is based on results from a 4-week study in children ages 6 to 11. The trial delivered results that were consistent with earlier research on the medicine.

UPDATED: Wednesday, October 4 at 9:20 a.m.

Novavax's updated COVID-19 vaccine has picked up an FDA authorization for use in people 12 and older.

The new vaccine is designed to better protect against variants that are currently circulating, the FDA said in a release.

People who have received a prior COVID vaccine can get one dose of the new Novavax shot as a booster, while unvaccinated people can get a two-dose series.

The FDA leveraged several information sources for the authorization, including manufacturing data, non-clinical immune response data, the agency's evaluation of the company's prior vaccine trials and postmarketing data.

Novavax's authorization follows recent FDA endorsements for Pfizer and Moderna's updated shots.
In another win for Novavax, the World Health Organization threw its weight behind a new malaria vaccine.

The vaccine, called R21/Matrix-M, features an antigen developed by the University of Oxford and Novavax's Matrix-M adjuvant.

R21/Matrix-M is now the second malaria vaccine recommended by WHO. The other is GSK's RTS,S/AS01, which won its WHO recommendation in 2021.
In addition to the malaria news, the WHO's Strategic Advisory Group of Experts on Immunization (SAGE) has recommended certain uses for Takeda's dengue vaccine Qdenga.

SAGE recommended the vaccine for children ages 6 to 16 in areas of high dengue burden, Takeda said in a release.

The vaccine should be used 1 to 2 years prior to the age of peak dengue incidence and hospitalization in a given region, according to the group's recommendation.

WHO will consider SAGE's recommendation before making a final decision on the vaccine.

UPDATED: Monday, October 2 at 8:50 a.m.

Apellis single-use, on-body injector for Empaveli has scored its FDA approval.

Empaveli is approved to treat paroxysmal nocturnal hemoglobinuria in adults, and the injector nod gives patients more flexibility in deciding where they want to receive their therapy, the company said in a release.

Apellis developed the Empaveli on-body injector in collaboration with Enable Injections.

The drug is administered twice per week. Aside from the injector nod, it was previously approved for subcutaneous self-administration.
Biogen's biosimilar to Roche's autoimmune drug Actemra has won its FDA approval.

To be branded as Tofidence, the biosimilar is approved to treat severely active rheumatoid arthritis, polyarticular juvenile idiopathic arthritis and systemic juvenile idiopathic arthritis, Biogen said in a release.

Tofidence becomes the first Actemra biosimilar to win FDA approval. Biogen will market the drug under a license agreement with Bio-Thera, which developed the biosim.

The precise launch timing remains unclear. In a release, Biogen said it is "currently evaluating the potential launch timeline" for the drug.

For its part, Roche has said Actemra biosimilars could launch in the second half of the year.

UPDATED: Thursday, September 28 at 10:30 a.m.

Akebia Therapeutics has refiled its oral HIF-PHI vadadustat with the FDA after receiving "clear direction" from the agency, the company's CEO John Butler said. After an FDA rejection last year, Akebia is seeking a narrower indication for the treatment of anemia due to chronic kidney disease (CKD) only in patients on dialysis.

The resubmission package also includes postmarketing data from "tens of thousands" patients in Japan, where the drug is approved under the brand name Vafseo.

Akebia expects a standard six-month review for this resubmission. If approved, vadadustat will go up against GSK's Jesduvroq, which is also allowed only in dialysis-dependent patients in the U.S.
Oncopeptides has decided not to pursue an expanded EU nod for the multiple myeloma drug Pepaxti for earlier lines of treatment.

The drug last year won approval in Europe to treat patients who have received at least three prior lines of therapy. Drug reviewers at the European Medicines Agency recently adopted a positive opinion on the drug's use as a third-line treatment based on data from the phase 3 OCEAN trial, which showed mixed results.

But an analysis of the "rapidly evolving multiple myeloma landscape and the market potential for Pepaxti in different scenarios" has led Oncopeptides to abandon the application, the company said.

In the U.S., Oncopeptides is fighting the FDA over market withdrawal of Pepaxti.

UPDATED: Tuesday, September 26 at 2:41 p.m.

On the heels of Japan approving Biogen and Eisai’s Alzheimer’s disease drug lecanemab, Eli Lilly has asked the country’s Ministry of Health, Labour and Welfare to sign off on its Alzheimer’s treatment donanemab.

While the drug was shown to slow progression of the disease by 35% after 18 months, it also carries the risk of brain swelling and bleeding. Lilly has requested approval of donanemab in the U.S. and Europe. Both lecanemab and donanemab target the amyloid protein, though lecanemab targets it at an earlier stage of build-up.
The European Commission has approved its first multiple sclerosis biosimilar, Sandoz’s Tyruko (natalizumab). The copycat, which was developed by Polpharma, can be used for all approved indications of its reference product, Biogen’s Tysabri. It is sanctioned as a disease-modifying therapy for those with highly active relapsing forms of MS.

Sandoz is the consumer health and generics business unit of Novartis and will soon be spun out by the parent company as an independent firm.
The European Commission has granted marketing authorization to AbbVie’s Tepkinly to treat relapsed or refractory diffuse large B-cell lymphoma (DLBCL) as a third-line option. It is the lone subcutaneous T-cell engaging bispecific antibody approved for the patient population in the EU.

In the U.S., where the drug is known as Epkinly, it was approved for third-line use in the same patient group in May of this year.
Regeneron and Sanofi’s application for Dupixent to treat children ages 1 to 11 with eosinophilic esophagitis (EoE) has been accepted by the FDA with a priority review. The regulator has assigned a target decision date of January 31, 2024.

In May of last year, Dupixent became the first approved treatment for the inflammatory condition, which narrows the esophagus and can prevent patients from swallowing. That approval covered people 12 and older.

The drug, which is approved for asthma, atopic dermatitis and chronic rhinosinusitis, generated sales of $8.7 billion last year.
Phathom Pharmaceuticals has filed a new drug application with the FDA for vonoprazan as a daily treatment for non-erosive gastroesophageal reflux disease (GERD), a heartburn condition that affects roughly 38 million in the U.S. The company expects an action date in the third quarter of 2024.

Vonoprazan is a first-in-class potassium-competitive acid blocker which excelled in a phase 3 trial of 772 participants, achieving primary endpoints for its 10 mg and 20 mg doses.
The CDC has recommended Pfizer’s respiratory syncytial virus (RSV) vaccine Abrysvo to be given to women who are 32 to 36 weeks pregnant to protect infants from birth to age 6 months.

In May, Abrysvo was approved by the FDA for adults 60 and older. Then last month, the U.S. regulator expanded the endorsement to pregnant women, leaving it up to the CDC to recommend its use. Story

UPDATED: Monday, September 25 at 9:34 a.m.

After Eisai and Biogen's first approval for lecanemab in July in the United States, Japan has become the second country to approve the drug.

Japan's Ministry of Health, Labour and Welfare approved the drug under the condition that the partners conduct a post-marketing study to gather more data on its real-world use. The regulators also flagged risks of a side effect called amyloid-related imaging abnormalities, so the drug's launch will be designed to minimize that risk, the partners said in a release.

Eisai and Biogen are co-promoting the drug in Japan, where it's approved to slow progression of mild cognitive impairment and mild dementia due to Alzheimer's disease.
Also in Japan, GSK's RSV vaccine Arexvy has won approval for use in adults 60 and older.

The vaccine becomes the first RSV shot to win an approval in Japan. The country is the first in Asia to approve the shot, as well.

Besides Japan, the vaccine has won approvals in the U.S., Europe, the U.K., and Canada.

Phase 3 trials of the vaccine enrolled more than 1,000 participants in Japan, GSK said.
Rounding out a busy stretch for Japanese regulators, Ardelyx and Kyowa Kirin's tenapanor has won an approval in the island country.

Tenapanor will be marketed in Japan as Phozevel and is approved for the improvement of hyperphosphatemia in adults with chronic kidney disease on dialysis.

In the U.S., Ardelyx is planning to launch in the fourth quarter of 2023. The company previously received a complete response letter in 2023.

The Japanese approval triggers $35 million in milestone and other payments to Ardelyx, the company said in a release.
After reports of patients losing vision after receiving altered forms of Roche's cancer drug Avastin, Pakistan's drug regulator has issued a public update about the situation.

The drug is approved to treat certain cancers, but it's often used off label for diabetic retinopathy or other ophthalmic conditions.

Pakistan's regulator said the situation involves altered, dispensed or diluted Avastin used in "non-sterile" conditions. Authorities raided the offices of Genius Advanced Pharmaceutical Services and have started legal proceedings, according to the update.

UPDATED: Wednesday, September 20 at 10:20 a.m.

The FDA has accepted Takeda's resubmission for eosinophilic esophagitis (EoE) candidate Eohilia. A decision is now expected in the first half of 2024, according to the Japanese pharma company.

Also coded TAK-721, Eohilia is an oral suspension formulation of the corticosteroid budesonide. The drug was rejected by the FDA in late 2021. This time, Takeda reanalyzed its clinical data and spoke with the FDA before the filing.

If approved, Eohilia would go up against Sanofi and Regeneron's injectable Dupixent, which became the first FDA-approved EoE treatment last May.
Merck's application for Keytruda, used in combination with chemoradiation therapy, is now under FDA priority review for the treatment of newly diagnosed, high-risk, locally advanced cervical cancer. The target decision date is Jan. 20, 2024.

It was only in July that Merck announced that the Keytruda regimen bettered chemoradiotherapy alone at preventing cancer progression or death in the phase 3 Keynote-A18 trial. Detailed data have yet to be revealed.

If approved, Keytruda would have a chance to change more than two decades of practice and become the first immunotherapy approved in this cervical cancer setting.
Following an FDA nod in June, Pfizer has nabbed the European Commission's go-ahead for its oral alopecia drug Litfulo to treat adults and children ages 12 years and above with severe disease.

In the phase 2b/3 Allegro trial, 13.4% of patients with at least 50% scalp hair loss saw 90% or more scalp hair coverage after 24 weeks of treatment with Litfulo, compared with 1.5% with placebo.

As is the case in the U.S., Litfulo's EU label is broader than that of Eli Lilly's Olumiant, which is only approved in adults.
The European Commission also approved Menarini's oral SERD drug Orserdu to treat postmenopausal women and men with ER-positive, HER2-negative, ESR1-mutated breast cancer after at least one line of prior therapy. The drug was also the first oral SERD to cross the FDA finish line back in January.

ESR1 mutations are acquired mutations that develop following endocrine therapy, and they are found in up to 40% of patients with ER+/HER2- breast cancer, according to Menarini.

Compared with standard endocrine monotherapy, Orserdu reduced the risk of progression or death by 45% in previously treated ER+/HER2- patients with ESR1 mutations in the phase 3 Emerald trial.

UPDATED: Monday, September 18 at 2:35 p.m.

The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recommended against renewing the conditional marketing authorization for GSK's multiple myeloma drug Blenrep.

GSK's drug won a conditional approval in Europe in 2020 for patients who've tried four other therapies. Subsequently, the medicine failed a phase 3 trial, leading GSK to pull an accelerated approval in the U.S. last year.

Now, the drug could be setting up for a similar outcome in Europe. After the CHMP recommendation, the European Commission has the final say on the drug's marketing authorization.
Also at the CHMP, Takeda's Adcetris picked up a label expansion recommendation.

Specifically, CHMP reviewers endorsed Takeda's drug in combination with doxorubicin, vinblastine and dacarbazine in adults with previously untreated CD30+ Stage III Hodgkin lymphoma.

The drug won its conditional approval in Europe in 2012 and now boasts 6 approvals in there, according to Takeda.
Those were just two of the decisions during a busy meeting of the CHMP. Elsewhere, Almirall's Ebglyss snagged an approval recommendation for the treatment of moderate to severe atopic dermatitis in patients 12 and older who are eligible for systemic therapy.

In all, CHMP signed off on 9 approval recommendations and endorsed 11 label expansions at its meeting last week.
In the U.S., meanwhile, RedHill Biopharma on Monday announced the FDA approval of a new dosing regimen for Talicia.

The FDA approved a three-times-per-day dosing regimen for the H. pylori drug, specifying that the doses should be taken at least 4 hours apart with food. Previously, patients had to take the drug every 8 hours with food.

UPDATED: Friday, September 15 at 9:33 a.m.

Idorsia's Quviviq has won the support of England's National Institute for Health and Care Excellence (NICE) for the treatment of certain patients with insomnia in England and Wales.

Specifically, NICE reviewers recommended the drug for patients who've suffered from insomnia for at least three nights per week for at least three months, and whose daytime functioning is considerably affected. To be eligible for the therapy, patients must have tried cognitive behavioural therapy for insomnia (CBTi), or not be eligible for CBTi.

With the recommendation, Quviviq is set to become the first dual orexin receptor antagonist available through NHS England.
Merck's decades-old cancer drug Temodar has received updated labeling from the U.S. FDA.

First approved in 1999, the drug's new label covers the adjuvant treatment of adults with newly diagnosed anaplastic astrocytoma, plus the treatment of adults with refractory anaplastic astrocytoma.

Another previously approved use for the drug, in glioblastoma, remains the same.

The FDA updated the drug's labeling under a pilot program called Project Renewal, which is setting out to refresh the labels of old cancer drugs with significant real-world use.
Europe's Committee for Medicinal Products for Human Use (CHMP) recommended Merck's Keytruda for the adjuvant treatment of certain adults with non-small cell lung cancer. The approval recommendation applies to those who are at high risk of recurrence following complete resection and platinum-based chemotherapy.
Separately, the CHMP recommended Oncopeptides' Pepaxti for earlier lines of treatment for patients with relapsed, refractory multiple myeloma.

Oncopeptides is seeking approval for Pepaxti to treat patients who've tried two other therapies. With the CHMP recommendation, the European Commission is now set to decide on the potential label expansion.

UPDATED: Wednesday, September 13 at 8:39 a.m.

The U.S. Centers for Disease Control and Prevention (CDC) on Tuesday recommended new COVID-19 boosters from Pfizer and Moderna for all people ages 6 months and older, NBC News reports. The recommendation follows the FDA's endorsement of the shots earlier this week.

With the new CDC recommendation, the shots should start to roll out across the U.S. in the coming days, according to the CDC.

Pfizer and Moderna's new shots target the Omicron XBB.1.5 variant. Nearly all circulating cases are related to that strain, according to U.S. officials.

The rollout comes amid a recent uptick in cases in the U.S., and ahead of a potential "tripledemic" of influenza, COVID-19 and respiratory syncytial virus, officials have warned.
China's BioRay Pharmaceutical has received approval for its biologic zuberitamab for the treatment of CD20-positive diffuse large B-cell lymphoma from the country's National Medical Products Administration (NMPA).

With the approval, BioRay gave its new cancer drug the trade name Anruixi. The drug turned in positive results in trials of various stages, the company said in a release, including in a head-to-head versus Roche's Rituxan.

Anruixi becomes the first domestically developed Class I anti-CD20 drug to be approved in China. It succeeds BioRay's Anruize as the company's "next major product."

BioRay is studying its new medicine in autoimmune diseases, as well.

UPDATED: Monday, September 11 at 10:28 a.m.

UCB's inflammatory disease medicine Bimzelx has picked up a pair of recommendations from England's National Institute for Health and Care Excellence (NICE).

In final draft guidance, the agency endorsed the med to treat patients with active psoriatic arthritis and axial spondyloarthritis. NICE plans to publish its final decisions on the medicine next month.

Bimzelx is already recommended as a treatment for certain patients in the U.K. with plaque psoriasis.

In the U.S., regulators last year rejected the drug because of issues with a required pre-approval manufacturing inspection. More recently, the FDA delayed its decision on the med's second application, pushing it to the third quarter of 2023.
Israel's BioLineRx has won FDA approval for Aphexda, which the company says is the "first innovation in stem cell mobilization for multiple myeloma" to be approved in the U.S. in a decade.

Aphexda is a combination of BioLineRx's motixafortide and filgrastim, which is also known as Neupogen from Amgen.

The drug is approved to mobilize hematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients with multiple myeloma.

The company plans to launch the product later this month.

UPDATED: Tuesday, September 5 at 8:56 a.m.

England's National Institute for Health and Care Excellence (NICE) has signed off on Chiesi's Elfabrio for the treatment of Fabry disease in adults.

Chiesi has agreed to provide the drug under a "commercial arrangement," NICE said in its final draft guidance (PDF). Before any discount, the drug's annual treatment cost is £118,187 for a patient of average weight, the agency said.

The drug has been on a regulatory winning streak this year, picking up approvals in Europe and the United States in May. Before that, the FDA previously rejected the drug in 2021.

Israel-based Protalix developed the drug and licensed its global commercialization rights to Chiesi in 2018.
Viatris has received a tentative FDA approval for a pediatric formulation of a three-drug treatment for HIV.

The agency approved the company's application for the combination of abacavir 60mg, dolutegravir 5mg and lamivudine 30mg tablets for oral suspension to treat HIV-1 infection in pediatric patients.

In a release, Viatris said the tentative approval "will help enhance access" to the treatment in low- and middle-income countries.

The company previously inked a licensing deal with the Medicines Patent Pool for pediatric dolutegravir and entered a separate agreement with ViiV Healthcare and the Clinton Health Access Initiative to produce and distribute the fixed-dose abacavir/dolutegravir/lamivudine combo.
After a recent approval recommendation from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP), Pfizer and BioNTech's new Omicron XBB.1.5-targeted COVID vaccine has won an approval in the United Kingdom.

The U.K.'s Medicines and Healthcare products Regulatory Agency approved the shot for people 6 months and older.

UPDATED: Wednesday, August 30 at 1:55 p.m.

Pfizer and BioNTech's new COVID-19 vaccine has gained an approval recommendation from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP).

After a final sign-off from the European Commission, the updated shot will be ready to ship across Europe, Pfizer said in a release.

The new vaccine is tailored to target the Omicron XBB.1.5 sublineage of the virus. Regulators in Europe recommended the shot for people 6 months and older regardless of prior COVID vaccination history.

The recommendation is based on preclinical data showing the vaccine "generates an improved response against multiple XBB-related sublineages," Pfizer said.
Roche's spinal muscular atrophy drug Evrysdi has gained a label expansion in Europe.

Specifically, the European Commission endorsed the drug to treat SMA in patients 2 months and younger. The drug was previously approved for babies older than 2 months old in Europe.

The drug is now available to treat SMA patients of all ages in Europe, Roche said.

UPDATED: Tuesday, August 29 at 10:40 a.m.

The U.K. has become the first country to approve a subcutaneous version of Roche's PD-L1 inhibitor Tecentriq.

Developed using Halozyme Therapeutics' Enhanze drug delivery technology, the under-the-skin version is approved for the same indications as its intravenous counterpart.

The subcutaneous version, administered under the skin in about seven minutes, "saves time for patients and helps conserve resources in healthcare systems," Roche said.

Before this approval, Chinese authorities in late 2021 approved a homemade subcutaneous PD-L1 antibody, envafolimab from 3D Medicines, Alphamab Oncology and Simcere Pharmaceutical.
Bristol Myers Squibb's Reblozyl has won a key FDA approval to move up the treatment ladder to control anemia in patients with myelodysplastic syndromes who haven't received a prior erythropoiesis stimulating agent (ESA). The new nod also covers patients regardless of ring sideroblast status.

BMS has pegged Reblozyl's peak sales potential at $4 billion, with a large part to come from the first-line use.

In the phase 3 COMMANDS trial, Reblozyl beat Amgen and Johnson & Johnson’s ESA Epogen/Procrit at helping more patients live free of transfusion.
The European Commission last week approved Pfizer's RSV vaccine Abrysvo to protect both infants through maternal immunization and older adults.

The EU nod came days after the FDA expanded the shot's indication to include the maternal use. Pfizer has applications for the vaccine, also known as RSVpreF, under review in Japan and Canada.
Merck has won a go-ahead from the European Commission for Keytruda, used alongside trastuzumab and chemotherapy, for newly diagnosed unresectable or metastatic HER2-positive gastric or gastroesophageal junction adenocarcinoma. The approval is limited to patients whose tumors express PD-L1.

Merck recently asked the FDA to convert an accelerated approval for Keytruda in that use to a full nod, but also to restrict the label to PD-L1-positive disease. The move came after data from the phase 3 Keynote-811 trial found a progression-free survival benefit for Keytruda but only in patients with PD-L1-positive tumors.

UPDATED: Thursday, August 24 at at 9:55 a.m.

Pfizer and Astellas' label expansion effort for Xtandi in a new prostate cancer indication has gained a speedy review. The FDA has granted Xtandi a priority review for the proposed use of treating non-metastatic castration-sensitive prostate cancer with high-risk biochemical recurrence (BCR), the companies said.

The application leverages results from the phase 3 EMBARK study. In the trial, Xtandi, plus the prostate cancer drug leuprolide, outperformed placebo and leuprolide on the primary endpoint of metastasis-free survival, demonstrating a 58% reduction in the risk of metastasis or death versus the control arm.

Biochemical recurrence can be an early sign that prostate cancer is returning or will spread, Astellas' head of oncology development, Ahsan Arozullah, M.D., said in a statement.

The FDA aims to act on the application during the fourth quarter of 2023, the partners added.
AstraZeneca has picked up a pair of new approvals in Japan.

In oncology, the company's Merck-partnered Lynparza won a Japanese approval, in combination with Johnson & Johnson's Zytiga and the corticosteroid prednisolone, to treat BRCA-mutated metastatic castration-resistant prostate cancer. The Phase 3 PROpel study supported the filing.

Also Thursday, AstraZeneca said its Soliris has gained a Japanese approval to treat certain pediatric patients with generalized myasthenia gravis. Soliris is the only targeted therapy for this patient population in Japan, AZ said.

AstraZeneca picked up Soliris in its $39 billion buyout of Alexion in 2020.
Elsewhere, England's National Institute for Health and Care Excellence (NICE) has recommended Amryt's Filsuvez to treat skin wounds associated with dystrophic and junctional epidermolysis bullosa.

The final guidance means that around 670 patients in England will benefit from the new treatment option, NICE said.

With the guidance, Filsuvez becomes the first drug for EB to be recommended by NICE.
While COVID-19 no longer represents a major business focus at Gilead Sciences, the company's antiviral Veklury has picked up a U.S. label expansion.

Thursday, the company said the FDA approved a new label endorsing the drug, with no dose adjustments, to treat COVID-19 in people with mild, moderate and severe hepatic impairment.

Veklury is the only COVID antiviral that's approved across all stages of liver disease, the company said.

UPDATED: Wednesday, August 23 at at 10:41 a.m.

Daiichi Sankyo’s Enhertu is now cleared for use in Japan for adults with unresectable advanced or recurrent non-small cell lung cancer (NSCLC) with HER2 mutations that has progressed following chemotherapy treatment.

Japan’s Ministry of Health, Labour and Welfare previously granted the med orphan drug designation for the indication. The new use makes the fourth for the drug in Japan in just over three years and the second approval in the country this year alone, head of Daiichi’s R&D in Japan Wataru Takasaki, Ph.D., noted in the company’s release.

In a phase 2 trial of 151 patients, the med achieved a confirmed objective response rate of 53.8%.
As Bristol Myers Squibb’s Opdivo eyes a newly diagnosed bladder cancer nod in the U.S., the European Commission (EC) granted it a new indication as a monotherapy in the adjuvant treatment of those 12 years of age and older with stage IIB or IIC melanoma who have undergone complete resection.

Now, the drug is the EU’s only PD-1 inhibitor that’s indicated for stages IIB, IIC, III and stage IV resected melanoma as an adjuvant treatment.

The recent approval was based on a phase 3 trial that proved Opdivo’s ability to reduce the risk of recurrence or death by 58% over placebo. Recurrence-free survival rates came out to 89% over 12 months, compared with placebos 79%.
Pfizer and Astellas’ Xtandi is on the way to a potential FDA approval in non-metastatic castration-sensitive prostate cancer with high-risk biochemical reoccurrence.

The agency blessed the med with priority review in that indication with a decision date over the fourth quarter of this year.

Xtandi’s application is based on a phase 3 trial of 1,068 patients in which the drug, combined with hormone agonist leuprolide, reduced the risk of metastasis or death by 58% over placebo. The companies are in talks with regulatory authorities in other countries to explore the potential indication.

UPDATED: Tuesday, August 22 at at 8:58 a.m.

After a Japanese public health panel recommended a marketing authorization for Eisai's Alzheimer's disease drug Leqembi, the medicine is set for an approval in the company's home country, Bloomberg reports.

As part of Japan's approval process, the health ministry solicits the panel's recommendations before making decisions on new medicines. The ministry usually follows its panel's guidance, according to the news service.

In a note Monday, Jefferies Japan analyst Stephen Barker said he expected the drug to secure government reimbursement at around $13,700 per year, according to Bloomberg.

Leqembi won a full approval in the U.S. early last month. The company is also seeking an approval in Europe but hasn't reached that milestone yet.
Johnson & Johnson's Talvey has been gaining momentum on both sides of the Atlantic. After the drug won an FDA approval earlier this month, it's now scored an approval in Europe.

Regulators in Europe granted a conditional marketing authorization for Talvey as a monotherapy for treating adults with relapsed and refractory multiple myeloma who have tried at least three other treatments. To be eligible for Talvey, patients must have tried an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody. They must also have demonstrated disease progression on the last therapy, J&J said.

The marketing authorization comes after a positive recommendation from the European Medicines Agency's Committee for Medicinal Products for Human Use last month.

A bispecific, Talvey is a novel GPRC5D-targeted therapy that works by attracting T cells to target blood cancer cells expressing GPRC5D.

UPDATED: Thursday, August 17 at 1:34 p.m.

Merck's Keytruda has gained an endorsement from England's National Institute for Health and Care Excellence (NICE) to treat cancers with microsatellite instability (MSI) or mismatch repair deficient (dMMR) mutations.

It's the first time NICE has blessed a cancer immunotherapy based on the results of a "basket trial," Pharmaphorum reports. Such trials enroll patients with a different tumor types but with specific mutations.

For Keytruda's new recommendation, NICE supports use of the drug to treat patients with previously treated endometrial, biliary, colorectal, gastric or small intestine cancers with MSI or dMMR mutations.

The endorsement will open the treatment to around 300 patients in England, according to NICE.
Amicus' Pompe disease combination therapy of Pombiliti and Opfolda has been cleared for use in the United Kingdom.

The U.K.'s Medicines and Healthcare products Regulatory Agency (MHRA) granted approval for the drugs to treat adults living with late-onset Pompe disease. Alongside the MHRA nod, the drugs have gained an endorsement from NICE, Amicus said.

The U.K. approval follows the launch of the combo treatment in the European Union in June.

Meanwhile, in the U.S., the FDA deferred action on Amicus' application back in October. In making that decision, the FDA cited an inability to conduct a required manufacturing inspection before it could grant the approval.
AbbVie's migraine drug Aquipta has gained an approval in Europe for the prophylaxis of migraine in adults who have four or more migraine days per month.

In the U.S., the drug is branded Qulipta. Also known as atogepant, the drug won its original U.S. approval back in 2021.

The EU nod is based on two phase 3 trials that showed Aquipta beat out placebo in reducing monthly migraine days in patients with episodic and chronic migraine.

UPDATED: Wednesday, August 16 at 8:58 a.m.

The FDA has been quite busy lately. Already this week, the agency signed off on four drug approvals or label expansions, including new cancer meds from Pfizer and Johnson & Johnson.

Pfizer's Elrexfio won an approval to treat patients with multiple myeloma who've tried at least four prior lines of therapy, while J&J's Akeega snagged an FDA nod in a subset of patients with metastatic castration-resistant prostate cancer.

Beyond those two Big Pharma companies, Delcath's cancer treatment Hepzato Kit won an FDA approval almost 10 years after its initial rejection at the agency. And Revance's Daxxify scored its first therapeutic indication in cervical dystonia.
Also at the FDA, Servier's Tibsovo picked up a priority review in its proposed use to treat patients with isocitrate dehydrogenase 1-mutated relapsed or refractory myelodysplastic syndromes.

The company's application is based on a small phase 1, open-label study that showed the drug induced a complete response in nearly 40% of patients.

Under the priority review program, the FDA seeks to act on an application within six months. Tibsovo is currently approved to treat certain patients with acute myeloid leukemia.
In China, Innovent Biologics' Sintbilo won approval (PDF) from the National Medical Products Administration.

Sintbilo is approved in China to treat adults with with primary hypercholesterolemia. It becomes the first China-developed PCSK9 inhibitor to win an approval in the country.

The NMPA based its approval on the results of two phase 3 trials.

UPDATED: Thursday, August 10 at 9:03 a.m.

Gavreto, the RET inhibitor from Blueprint Medicines that Roche paid a hefty sum to get its hands on back in 2020, has converted an accelerated approval to a full FDA nod.

Specifically, the FDA approved the drug to treat adults with metastatic RET fusion-positive non-small cell lung cancer (NSCLC), as detected by an FDA-approved test. This follows the med's accelerated approval in the use back in September of 2020.

Still, this approval comes at a precarious time for the drug. Just a month ago, Roche pulled Gavreto's accelerated approval in advanced RET-mutant medullary thyroid cancer, citing the infeasibility of running a confirmatory trial.

And before that, Roche in February decided to terminate its Blueprint partnership altogether and return rights to the medicine to its developer. The companies are in a transition period and will fully end their collaboration in February.
Johnson & Johnson's Talvey has picked up an FDA approval to treat adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of treatment.

With the nod, Talvey becomes a first-in-class bispecific antibody for multiple myeloma treatment. The drug is a bispecific T-cell engaging antibody that targets the CD3 receptor on the surface of T cells, plus the G protein-coupled receptor class C group 5 member D (GPRC5D) on the surface of certain other cells, according to J&J.

Talvey is given weekly or every other week as a subcutaneous injection after a start-up period. It's also on track for an approval in Europe after recently scoring backing from officials at the European Medicines Agency.

UPDATED: Tuesday, August 8 at 9:37 a.m.

Following the European Commission's (EC) recent decision to revoke the conditional marketing authorization for Adakveo in Europe, Novartis says it's abiding and pulling the drug from the market there.

The drug scored its European approval back in 2020 to prevent recurrent vaso-occlusive crises, or pain crises, in sickle cell disease patients ages 16 and older.

But then in the phase 3 STAND trial, Adakveo couldn’t reduce the number of painful crises leading to a healthcare visit compared with placebo. That led a regulatory committee to recommend the market pull back in May.

Last week, the EC adopted that decision.

The drug remains approved in the U.S., and Novartis said it "continues to discuss the STAND study results with the US FDA and other health authorities globally.
GSK's new respiratory syncytial virus (RSV) vaccine, Arexvy, continues to pick up approvals.

Officials in Canada have approved the shot for people ages 60 and older. With the approval, GSK's vaccine becomes the first RSV shot for older adults in Canada.

GSK said it expects the vaccine to be available in Canada before the fall and winter RSV season.

UPDATED: Thursday, August 3 at 3:46 p.m.

Sanofi and AstraZeneca’s respiratory syncytial virus (RSV) antibody for infants, Beyfortus, scored a crucial recommendation from the Centers for Disease Control and Prevention's (CDC) Advisory Committee on Immunization Practices (ACIP).

Thanks to an unanimous vote, the preventative antibody shot is set to be added to the U.S.’s children’s vaccine schedule .

The shot has a recommended list price of $495 and is expected to roll out ahead of the upcoming 2023-2024 RSV season.

“We appreciate the FDA and CDC leadership, as well as the ACIP public health experts, for recognizing and quickly acting on the threat RSV poses to all infants,” Sanofi's vaccines head Thomas Triomphe said in a statement.

UPDATED: Thursday, August 3 at 9:20 a.m.

After being approved in adults for more than 3 years, Merck's Zaire ebolavirus vaccine Ervebo has picked up an FDA approval to be used in children 12 months and older.

This approval comes after the shot's initial endorsement by the regulator in December 2019.

Meanwhile in Europe, the vaccine recently earned backing for use in kids as young as 1 year old from the Committee for Medicinal Products for Human Use (CHMP). The vaccine looks to be on track for an EU approval in this population in the coming months.

In a statement, Merck stressed that its vaccine "does not protect against other species of Ebolavirus or Marburgvirus and the duration of protection conferred by Ervebo is unknown."
Taiho Oncology is expanding the list of indications for its 8-year-old cancer drug Lonsurf.

As opposed to the drug's single-agent use, this new approval covers the medicine in combination with bevacizumab, or Roche's Avastin, for use in previously treated metastatic colorectal cancer

In a late-stage study, the combo proved to be more efficacious than Taiho’s drug alone. Compared with Lonsurf monotherapy, the combination reduced the risk of death by 39% in a specific group of previously treated patients.

This latest approval comes as Taiho's drug nears a clash with Takeda and Hutchmed’s fruquintinib in colorectal cancer.

UPDATED: Monday, July 31 at 9:04 a.m.

The FDA has approved a second naloxone spray for over-the-counter sales in the United States. Nonprofit Harm Reduction Therapeutics has won the agency's backing for RiVive naloxone spray.

RiVive's approval advances Harm Reductions' "mission to prevent opioid overdose deaths by making free or low-cost over-the-counter naloxone available to everyone across the United States," the company said in a statement on its website.

Harm Reduction aims to make the drug available by early 2024.

This development follows Emergent BioSolutions' groundbreaking Narcan OTC approval in March.
After a May 2023 recommendation from Europe's Committee for Medicinal Products for Human Use (CHMP), Marinus Pharmaceuticals' Ztalmy has won a full approval in the bloc.

The drug is approved for the adjunctive treatment of epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients ages 2 to 17, the company said. Patients can continue on the treatment after they turn 18.

Ztalmy's EU approval was supported by phase 3 data showing that the drug was associated with a 30.7% reduction in 28-day major motor seizure frequency compared with a 6.9% reduction for those receiving placebo.

Under a July 2021 deal, Orion Corporation acquired rights to commercialize the drug in Europe.

UPDATED: Friday, July 28 at 9:27 a.m.

After a positive opinion from Europe's drug reviewers last month, AstraZeneca's Soliris has officially clinched approval there to treat children and adolescents with refractory generalised myasthenia gravis (gMG).

The European Commission has approved the drug to treat children ages 6 to 17 who have refractory gMG and who have anti-acetylcholine receptor (AChR) antibodies. With the nod, Soliris is the first targeted therapy for pediatric gMG patients in Europe.

Generalised myasthenia gravis is a rare and severe neuromuscular disease that leads to loss of muscle function and weakness.
Also in Europe, Gilead's Trodelvy has picked up an approval for certain patients with pre-treated HR+/HER2- metastatic breast cancer.

Specifically, the drug is approved as a monotherapy for patients with unresectable or metastatic HR+/HER2- breast cancer who have received endocrine-based therapy, plus at least two additional systemic therapies in the advanced setting.

Gilead's TROPiCS-02 study supported the approval. In the trial, Trodelvy posted an overall survival advantage of 3.2 months versus a single-agent chemotherapy of a physician's choice. The drug also delivered a 34% reduction in risk of disease progression or death.

Gilead's drug scored a similar approval in the U.S. earlier this year.

UPDATED: Wednesday, July 26 at 9:15 a.m.

With its U.S. sales suffering from generic competition, Amarin has turned to Europe for its launch of fish-oil-based cardio drug Vazkepa. Now, the company has picked up national reimbursement in Spain.

Spain's Drug Pricing Committee has recommended the drug to reduce the risk of cardiovascular events in patients with high cardiovascular risk. Cardiovascular disease is the leading cause of death in Spain, according to figures shared by Amarin.

The development comes about a week after Amarin said it would lay off about 120 of its 385 staffers. The company also named Patrick Holt its new CEO last week. Holt replaced Karim Mikhail, who stepped aside earlier this year as the company faced activist investor pressure from Sarissa.
Eli Lilly and Boehringer Ingelheim's SGLT2 inhibitor Jardiance has gained a European approval to treat adults with chronic kidney disease (CKD).

The drug stands to advance the standard of care for some of the tens of millions of people in Europe living with CKD, the companies said in a release. It could also cut healthcare system burdens by cutting hospitalization rates among these patients. Jardiance already carried European approvals in type 2 diabetes in heart failure.

The European approval comes ahead of a potential FDA nod in CKD. Early this year, the FDA accepted the partners' application for Jardiance as a CKD treatment. The agency is set to make a decision on that application this year.
After a positive—yet limited—decision by Europe's Committee for Medicinal Products for Human Use (CHMP), GSK has decided not to advance its application in the bloc for oral anemia drug daprodustat.

In its second-quarter earnings release, GSK said it's pulling the filing because of a "significant reduction" in the size of the drug's commercial opportunity after CHMP members backed the drug only in CKD patients who are on dialysis. The panel did not support the drug's use in patients who are not on dialysis.

In the U.S., the drug is approved as Jesduvroq in adult CKD patients who have been on dialysis for at least four months.
Well underway with its U.S. launch, Mirum Pharmaceuticals said its liver disease drug Livmarli has picked up an approval in Canada.

Regulators in Canada endorsed the drug to treat cholestatic pruritus in patients with Alagille syndrome. The drug is already approved in this indication in U.S. and Europe, and Mirum is seeking an additional FDA approval for the med to treat cholestatic pruritus associated with progressive familial intrahepatic cholestasis.

UPDATED: Tuesday, July 25 at 8:58 a.m.

About a month after Pfizer won FDA approval for its severe alopecia areata drug Litfulo, the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion on the medicine.

The decision means the drug will likely gain a full European approval in the coming months.

Litfulo, a kinase inhibitor, won an FDA approval in late June as a treatment for severe alopecia areata in patients 12 and older.
Also at CHMP, Johnson & Johnson's bispecific antibodies Talvey and Tecvayli picked up endorsements to treat certain patients with relapsed and refractory multiple myeloma, the drugmaker said late last week.

Specifically, CHMP backed Talvey as a monotherapy for patients who've had disease progression after three prior therapies, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody.

Meanwhile, the committee recommended Tecvayli under a "reduced, biweekly dosing schedule of 1.5mg/kg every other week in patients who have achieved a complete response or better for six months or longer," J&J added. The drug had already been approved at a higher dose since last summer.

Multiple myeloma remains incurable despite scientific advances, and patients continually need new treatment options, J&J noted.
Europe's drug watchdog further signed off on approval recommendations for three biosimilars.

Sandoz's biosimilar of Biogen's multiple sclerosis drug Tysabri, also known as natalizumab, gained a CHMP endorsement. If approved by the European Commission (EC), it would be the first Tysabri biosim in the bloc.

In addition, Biocon Biologics' biosimilar of Bayer and Regeneron's star eye drug Eylea gained a CHMP nod. The EC is expected to decide on the application by the end of September, Biocon Biologics said.

Lastly, Fresenius Kabi said CHMP supported approval for its biosimilar of Roche's immunology drug Actemra.

UPDATED: Friday, July 21 at 10:57 a.m.

Merck's gefapixant, an investigational drug for refractory or unexplained chronic cough, has gained the support of the European Medicines Agency's Medicinal Products for Human Use (CHMP).

This endorsement likely sets the drug up for an official from the European Commission later this year.

The CHMP based its opinion on the COUGH-1 and COUGH-2 trials. In both studies, the drug showed a statistically significant reduction in 24-hour cough frequency versus placebo.

The drug was rejected by the U.S. FDA back in January.
Also at the CHMP, Bristol Myers Squibb's Opdivo scored a positive opinion as an adjuvant treatment for patients with completely resected stage IIB or IIC melanoma.

In the trial supporting this recommendation, Opdivo showed a statistically significant and clinically meaningful benefit in recurrence-free survival versus placebo.

Previously, Opdivo won a European approval for the adjuvant treatment of adult patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection.
On the same day that Ipsen received a positive CHMP recommendation for Bylvay as treatment for Alagille syndrome (ALGS), it also got a thumbs down from Europe’s Committee for Orphan Medicinal Products (COMP) to retain Bylvay’s orphan drug designation in the indication.
Ipsen, which acquired Bylvay in a $952 million buyout earlier this year of Albireo, said it will appeal the decision, which comes a month after the FDA approved Bylvay in ALGS.

Ipsen is in competition with Mirum Pharmaceuticals to treat three primary pediatric liver diseases. Mirum's drug, Livmarli, already has an approval in Europe and the U.S. to treat ALGS.
Spanish plasma specialist Grifols has wrapped up a successful phase 4 trial in the United States for biweekly dosing of Xembify in patients with primary immunodeficiencies (PIDs), setting up a potential label extension.

The subcutaneous immunoglobulin product was blessed by the FDA in 2019 as a weekly dose. Europe has already signed off on Xembify’s biweekly regimen.

UPDATED: Wednesday, July 19 at 1:33 p.m.

After a negative opinion in May by regulators at the European Medicines Agency, Ipsen has announced that palovarotene, a proposed treatment for the ultra-rare disease fibrodysplasia ossificans progressiva (FOP), has been rejected by the European Commission.

Ipsen said it will continue to seek approvals in other countries and regions. In the U.S., the drug is under review at the FDA, with an action date set for August 16.

The drug is already authorized in Canada for appropriate patients and in the U.A.E. under a provisional pathway. It's marketed in those countries as Sohonos.

Previously in the drug's development path, palovarotene suffered an FDA rejection and weathered prior delays at the agency. Ipsen acquired the drug in its $1.31 billion purchase of Clementia Pharmaceuticals in 2019.

UPDATED: Tuesday, July 18 at 9:30 a.m.

Sixteen months after getting a rejection from the FDA for its anemia drug vadadustat, Akebia Therapeutics plans to resubmit its new drug application for the treatment by the end of the third quarter, the company said.

Akebia has completed an End of Dispute Type A meeting with the regulator which was “informative and productive,” CEO John Butler said.

Akebia expects to receive the meeting’s minutes by mid-August and will then resubmit its application for vadadustat in adult patients with anemia due to chronic kidney disease who are on dialysis.

Vadadustat is approved in 34 countries but when it was rejected in March of 2022 for safety concerns including thrombeombolic events, Butler then expressed “extreme disappointment and frustration.” Less than two months later, Akebia’s commercial partner on the drug, Otsuka, backed out of a licensing deal to market it in the U.S. and Europe.
The FDA has given a thumbs-up to AstraZeneca and Sanofi’s antibody Beyfortus (nirsevimab) as a preventive for RSV-associated lower respiratory tract disease in babies.

The approval covers newborns and infants born during or entering their first RSV season as well as kids up to 24 months old who remain at risk of RSV through their second disease season. Europe signed off on the drug in November of last year.

The go-ahead thrusts Sanofi and AZ into the new RSV market. Earlier this summer, Pfizer and GSK won back-to-back FDA approvals for their adult vaccines. In Pfizer’s case, the company is also working on a vaccination to protect babies through maternal immunization. The FDA is set to make a decision on that application next month. Story

UPDATED: Monday, July 17 at 11:15 a.m.

Elevar has taken another step toward potential approval of its combination therapy for unresectable liver cancer as the FDA has accepted the new drug application for its investigational VEGFR inhibitor rivoceranib plus Jiangsu Hengrui Pharma’s PD-1 inhibitor camrelizumab, the company said on Monday. The agency has set a decision date for May 16, 2024.

The combo was approved for first-line use in China in February and now is set up for a potential market battle with Roche’s PD-L1/VEGF combo of Tecentriq and Avastin. The Roche duo is the only immunotherapy approved in the U.S. for front-line liver cancer.

Paving the way for the application are results from a phase 3 trial which showed that the rivoceranib-camrelizumab combo slashed the risk of death by 38% over Bayer’s old standard Nexavar. Patients who took the combo lived a median 22.1 months, versus 15.2 months for the Nexavar group.

This marks an important step for another attempt to introduce a China-made PD-1 inhibitor to the U.S. market.
Gilead has gained an FDA green light for its COVID-19 antiviral Veklury to be provided to patients with kidney disease, including those on dialysis.

It becomes the only approved COVID antiviral for people with all stages of kidney disease in the U.S. The therapy was approved in Europe for the same patients three weeks ago.

More than 1 in 7 people in the U.S. have kidney disease and are at an increased risk of dying from COVID, Gilead said.

The approval was based on two studies that demonstrated the pharmacokinetics and safety profile of Veklury in those with kidney disease, with no new safety signals observed.

UPDATED: Tuesday, July 11 at 2:30 p.m.

Less than a month after Roche's Columvi won its inaugural FDA approval, the drug has done the same in Europe.

The European Commission has signed off on Columvi, also known as glofitamab, to treat adults with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy. The European nod comes almost a month after the med's FDA approval to treat DLBCL, plus large B-cell lymphoma arising from follicular lymphoma, also after two prior treatments.

Columvi is now the first CD20xCD3 T-cell-engaging bispecific antibody in Europe to treat the most common and aggressive form of lymphoma, Roche said in a statement. The drug's European approval was supported by phase 1/2 data showing it induced early and long-lasting complete responses in DLBCL patients who had tried several other lines of therapy.
French pharma giant Sanofi has picked up a pair of regulatory wins in different parts of the world.

First, the company's chronic graft-versus-host disease (cGVHD) drug Rezurock is set to launch in Scotland after an endorsement by the Scottish Medicines Consortium (SMC), PharmaTimes reports. SMC blessed the drug based on data showing that three-fourths of cGVHD patients who received the treatment experienced a response within a year. In addition, 7 out of 126 patients in the trial experienced a complete response in all of their affected organs, according to PharmaTimes.

Also for Sanofi, the company's star immunology drug Dupixent has gained an approval in India to treat atopic dermatitis.

With the approval, Dupixent becomes the first biologic in India for patients with moderate-to-severe atopic dermatitis who don't achieve adequate control with other prescription options.

UPDATED: Monday, July 10 at 4:00 p.m.

Novartis’ Leqvio, which was approved by the FDA in December of 2021 to reduce low-density lipoprotein cholesterol (LDL-C), has picked up another indication that will broaden its use in the U.S. The treatment will now be available to patients with primary hyperlipidemia (high LDL-C).

It also can be used earlier in LDL-C treatment, added to diet and statin therapy, for those who have not had a cardiovascular event but at are an increased risk of heart disease.

Leqvio, which generated sales of $112 million last year, is the only small interfering RNA (siRNA) therapy to reduce LDL-C. It is administered twice a year after two introduction doses. Its label expansion was facilitated by a trial that showed it lowered LDL-C by up to 52% versus placebo in patients with heart disease or at increased risk of heart disease who couldn’t reach their LDL-C target with statin therapy.
Two months after winning approval in the United States for of its respiratory syncytial virus (RSV) vaccine in people 60 and older, GSK has earned the same sanction on its home turf.

The U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) has signed off on Arexvy. It becomes the first RSV vaccine for people ages 60 and older in England. Six weeks ago, Pfizer won approval from the FDA for its RSV shot Abrysvo in those 60 and older.
GSK’s Jemperli has yet to receive marketing authorization in the U.K., but eligible endometrial cancer patients will be able to receive it through the Early Access to Medicines Scheme (EAMS).

The program allows patients with life-threatening conditions to receive unapproved drugs where there is an unmet need. The anti-PD-1 therapy is taken in combination with platinum-containing chemotherapy for those with mismatch repair deficient/microsatellite instability-high advanced or recurrent endometrial cancer who are eligible for systemic treatment.

Endometrial cancer is the most common gynecological cancer in the U.K., according to PMLiVE. Patients with an advanced form have a poor prognosis.

UPDATED: Friday, July 7 at 8:22 a.m. ET

At long last, Novavax has scored full approval for its COVID-19 vaccine in Europe.

Friday, the company said the European Commission granted full approval for Nuvaxovid when used as a primary series in people 12 and older and as a booster in people 18 and older.

Still, it's unclear how much demand Novavax will see for its shot in Europe, where mRNA shots have long been the top choice. For all of 2023, Novavax expects to generate between $1.4 billion and $1.6 billion from its COVID shot.
As Alnylam works to expand the reach of its Onpattro, the company has revealed the date for an upcoming advisory committee meeting.

On September 13, external experts from the FDA's Cardiovascular and Renal Drugs Advisory Committee will review the company's application for Onpattro to treat cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis.

If Onpattro scores an approval in the proposed use, the drug would go up against Pfizer's blockbuster duo of Vyndaqel and Vyndamax.

Meanwhiel, Onpattro already carries an approval to treat polyneuropathy of hereditary ATTR amyloidosis in adults.
Incyte's Opzelura has been granted an approval in the U.K. to treat non-segmental vitiligo with facial involvement in people 12 and older.

With the approval from the Medicines and Healthcare Products Regulatory Agency, Opzelura becomes the first treatment in the U.K. to address repigmentation in non-segmental vitiligo.

Patients with vitiligo experience skin depigmentation because of the body's destruction of pigment-producing cells called melanocytes. About 1 in 100 people in the U.K. will suffer from vitiligo, according to Incyte, with most of them having non-segmental vitiligo.

UPDATED: Tuesday, June 27 at 9:20 a.m. ET

Amicus Therapeutics has won EU approval for both components of its Pompe disease combination. After a March go-ahead for Pombiliti, the European Commission has cleared Opfolda, or miglustat, the company said Tuesday.

Once consumed into the blood, Opfolda stabilizes Pombiliti, which is a long-term enzyme replacement therapy. The two drugs are meant to be taken as a combo for adults with late-onset Pompe disease.

Amicus said it will immediately launch the meds in Germany and is starting reimbursement talks with healthcare authorities in Europe.

In the U.S., the FDA is reviewing the regimen, with a decision expected in the third quarter of 2023, Amicus said.
Merck and partner Ridgeback Biotherapeutics have withdrawn their EU application for COVID-19 antiviral molnupiravir, known in the U.S. as Lagevrio, Reuters reports.

Drug reviewers at the European Medicines Agency had in February declined to back the drug for COVID, and Merck later requested a re-examination.

"We are evaluating our options on how we can generate additional evidence supportive of Lagevrio for the treatment of COVID-19," Dean Li, M.D., Ph.D., president of Merck Research Laboratories, said in a statement.
The FDA has lifted a clinical hold on Pharvaris' deucrictibant for the on-demand treatment of hereditary angioedema (HAE), but a halt on the drug's development as a prevention therapy remains in place.

The FDA slammed on the brakes in August and requested that Pharvaris conduct an additional long-term rodent toxicology study before continuing with clinical trials.

The FDA has now removed the hold for the on-demand treatment use based on an interim analysis of the nonclinical study, Pharvaris said. It plans to have the full, 26-week nonclinical data by the end of 2023 to resolve the remaining hold on the prophylactic element.

The company has an immediate-release capsule and an extended-release tablet of deucrictibant in mid-stage global clinical trials. The drug is modeled after Takeda's HAE med Firazyr.
Bristol Myers Squibb has received the European Commission's blessing for Camzyos, the obstructive hypertrophic cardiomyopathy therapy that the company got from its $13.1 billion MyoKardia acquisition.

Camzyos got its FDA nod last April. BMS hopes the first-in-class drug could rake in more than $4 billion sales at peak.

UPDATED: Friday, June 23 at 10:00 a.m. ET

The European Medicines Agency's drug reviewers have formally adopted a negative opinion against the approval of Amylyx Pharmaceuticals' amyotrophic lateral sclerosis (ALS) therapy, known as Relyvrio in the U.S. and Albrioza outside the U.S.

Amylyx had said in May that the EMA was "trending toward a negative opinion" on the drug's application.

In snubbing Amylyx, the EMA said it had concerns that the main CENTAUR study "did not show convincingly that Albrioza was effective in slowing down the worsening of the disease." The agency questioned how the survival data were collected and analyzed, calling them "not reliable."

In response, Amylyx said it will request a formal re-examination of the application, a process that will take about four months.
Meanwhile, the EMA has recommended the approval of AbbVie's CGRP inhibitor Aquipta, known as Qulipta in the U.S., for the prevention of migraine in adults who have at least four migraine days per month.

Last April, Pfizer's rival oral CGRP drug Nurtec received its own European approval but in a slightly different population. Nurtec's approval covers treatment of acute migraine, but its prevention indication only includes episodic migraine in patients who have at least four migraine attacks per month.

If approved, Aquipta's prevention label will include chronic migraine, which is characterized by 15 or more headache days a month and at least eight migraine days.
Moderna on Thursday said it has filed an application with the FDA for its updated COVID-19 vaccine to target the XBB.1.5 variant.

The move comes a week after an FDA advisory committee voted unanimously that new COVID shots for this coming season should be monovalent and target XBB.1.5.

In a statement on Thursday, Moderna CEO Stéphane Bancel said early clinical data showed the updated Spikevax vaccine is effective in generating an immune response against the current XBB variants of concern.

The mRNA company said it will deliver updated doses in time for the fall vaccination season.
The EMA has also adopted a positive opinion on GSK's Jesduvroq but only for patients with chronic kidney disease-anemia who are on chronic dialysis.

In February, Jesduvroq became the first—and so far still the only—in the oral HIF-PHI drug class to win an FDA approval, although it's also only for dialysis-dependent patients.

Akebia Therapeutics' Vafseo got its EU go-ahead in dialysis-dependent patients in April. Thanks to an EU approval in 2021, FibroGen and Astellas' Evrenzo is allowed in both dialysis and nondialysis patients. But Astellas recently took an impairment over the drug because of depressing sales outlook.

UPDATED: Wednesday, June 21 at 9:30 a.m. ET

The FDA has approved Eli Lilly and Boehringer Ingelheim's Jardiance and Synjardy to treat type 2 diabetes in children.

Specifically, the agency approved the drugs as adjuncts to diet and exercise to improve blood sugar control in kids 10 and older with type 2 diabetes.

Both drugs contain the active ingredient empagliflozin, while Synjardy also includes the popular diabetes drug metformin.

Jardiance won its original FDA approval in 2014 and Synjardy followed in 2015. Both of those nods were for the adult patient population.

The FDA made the latest endorsement based on a study showing patients aged 10 to 17 fared better on blood sugar control when treated with empagliflozin compared to placebo.
Bluebird bio's FDA application for sickle cell disease gene therapy prospect lovotibeglogene autotemcel has garnered an FDA priority review.

The priority review sets the drug up for a December 20, 2023 decision date at the FDA.

This development comes after bluebird submitted the application in April after several delays.

Last year, bluebird won a pair of gene therapy approvals in the U.S., enabling it to kick off a transformation into a commercial gene therapy company in the U.S.
Argenx has won an FDA approval for a subcutaneous version of its generalized myasthenia gravis drug Vyvgart.

The FDA approved the subcutaneous drug for adults who are anti-acetylcholine receptor (AChR) antibody-positive, a group that represents about 85% of the total gMG patient population, according to argenx.

With the approval, Vyvgart Hytrulo becomes the first subcutaneous injectable for gMG. This approval follows Vyvgart's original approval in late 2021.

The new product uses Halozyme's Enhanze drug delivery tech.
The FDA has converted an accelerated approval for Amgen's Blincyto in minimal residue disease (MRD)-positive B-cell precursor acute lymphoblastic leukemia (ALL) into a full approval.

The original approval was granted in 2018 based on MRD remission data from a single-arm trial. Amgen submitted two phase 3 trials to get the full nod.

A collaborative group-conducted phase 3 study recently showed that Blincyto could work in MRD-negative ALL as well.
Calliditas Therapeutics is seeking a full FDA approval for its rare disease drug Tarpeyo.

The biotech has submitted a supplemental drug application seeking a full approval for the medicine to reduce proteinuria in adults with primary IgA nephropathy at risk of rapid disease progression.

Tarpeyo won an accelerated FDA approval December 2021 and is the first treatment in this indication.

Calliditas is also working with its partner in Europe, Stada, on a potential full approval there.

UPDATED: Wednesday, June 14 at 9:45 a.m. ET

Acadia has selected a drug called ACP-101, or intranasal carbetocin, to advance into a phase 3 study in Prader-Willi syndrome (PWS).

The company recently met with the FDA and "reached alignment to further evaluate the 3.2 milligram dose of ACP-101 in a pivotal Phase 3 study,” Acadia's head of R&D, Doug Williamson, said in a statement.

Acadia acquired rights to develop the drug with its purchase of Levo Therapeutics last year. In an FDA presentation (PDF) from 2021, Levo said PWS is a "rare, life-threatening neurodevelopmental genetic disorder" that affects 8,000 to 10,000 people in the U.S.

As for carbetocin, it's a decades-old medicine that has been used to prevent excessive bleeding after childbirth. In its 2021 presentation, Levo said the drug is safe and well tolerated, and that it "provides clinically meaningful benefits to patients with PWS."

If the phase 3 trial is successful, Acadia would seek an FDA approval, Williamson added.
Ironwood Pharmaceuticals' Linzess has won FDA approval to treat functional constipation in pediatric patients ages 6 to 17 years old. Linzess is now the only medicine carrying this approval in the pediatric patient population.

The FDA nod came after the drug won a priority review for the review earlier this year. The FDA based its approval on the results of a phase 3 study in 328 patients.

Since 2012, the drug has been available to treat adults with irritable bowel syndrome with constipation or chronic idiopathic constipation.

Ironwood is partnered with AbbVie on Linzess development and marketing in the United States.

UPDATED: Monday, June 12 at 10:15 a.m. ET

Phathom's resubmission for vonoprazan in erosive gastroesophageal reflux disease (GERD) has been accepted by the FDA, the company said on Monday.

The development comes after the agency issued a complete response letter back in February requesting the company to generate more stability data to ensure that levels of a potential cancer-causing agent in the drug remain at or below the daily acceptable intake limits.

The new filing contains extra stability data to support the drug's shelf life, Phathom said. The agency is now set to decide on this application by November 17.

Vonoprazan previously won an FDA approval to treat Helicobacter pylori and is branded as Voquezna in that use.

If Phathom's new application is approved, the company plans a dual launch for the drug in erosive GERD and Helicobacter pylori during the fourth quarter of 2023.
AstraZeneca's FDA application for a combination of Faslodex and investigational agent capivasertib has been granted a priority review.

Faslodex already carries FDA approvals in combination with CDK4/6 inhibitors to treat HR-positive, HER2-negative metastatic breast cancer whose disease has progressed after antiestrogen therapy.

For this combination, AstraZeneca is also proposing adding the first-in-class AKT inhibitor capivasertib to treat patients with HR-positive, HER2-negative locally advanced or metastatic breast cancer after recurrence or progression on an endocrine-based regimen.

The FDA is due to make a decision on the filing during the fourth quarter of 2023.

Supporting the filing are data from the phase 3 CAPItello-291 trial. In the trial, the combo demonstrated a 40% reduction in the risk of disease progression or death versus Faslodex and placebo.

UPDATED: Friday, June 9 at 8:30 a.m. ET

AstraZeneca and Merck's Lynparza has received a positive recommendation from England's National Institute for Health and Care Excellence for the treatment of BRCA-mutated, relapsed, platinum-sensitive high-grade ovarian, fallopian tube or peritoneal cancer.

The approval covers patients who have had two or more courses of platinum-based chemotherapy.

The NICE endorsement comes right after Lynparza picked up a limited FDA approval in metastatic castration-resistant prostate cancer in the United States. Specifically, the FDA last week approved the drug to treat patients with mCRPC whose tumors have BRCA mutations. That limits the drug's reach in mCRPC to about 10% of the patient population.

This recommendation follows one early last month for the drug to treat certain patients with HER2‑negative high-risk early breast cancer.

UPDATED: Thursday, June 8 at 2:45 p.m. ET

The FDA has accepted Merck’s application for blockbuster Keytruda combined with standard-of-care chemotherapy (gemcitabine plus cisplatin) to treat biliary tract cancer (BTC).

The supplemental Biologics License Application is backed by results from the phase 3 KEYNOTE-966 trial of 1,069 patients, which showed that Keytruda provided a statistically significant improvement in extending survival compared to chemo alone.

The agency will decide on the label expansion by February 7, 2024. The combo would cover patients with locally advanced unresectable or metastatic BTC. Among liver cancers, BCT accounts for roughly 15% of cases. Approximately 211,000 people are diagnosed with BTC each year, Merck said.
Thirteen months after UCB received a complete response letter (CRL) from the FDA for its application for bimekizumab, the Belgium-based company has scored two label expansions for the IL-17A and IL-17F inhibitor in Europe. The European Commission has endorsed Bimzelx to treat psoriatic arthritis (PsA) and active axial spondyloarthritis (axSpA).
Two years ago, the treatment was approved in Europe for plaque psoriasis. But in May of 2022, it was rejected by the FDA, which cited the need for pre-approval inspection observations to be resolved. The FDA accepted UCB’s resubmission in December and is expected to make a decision in the coming weeks.
Five weeks after GSK beat two rivals to secure a world-first approval for its adult respiratory syncytial virus (RSV) vaccine in the U.S., it has done the same in Europe. The endorsement for Arexvy covers adults 60 and older in 30 European countries and is based on a phase 3 study that showed efficacy of 83% overall and 95% in older adults with at least one underlying medical condition.

The approval came earlier than expected and just two months after a thumbs up from the Committee for Medicinal Products for Human Use (CHMP). It will allow GSK to launch ahead of the fall RSV season. Roughly 270,000 elderly people are hospitalized each year for RSV with 20,000 deaths, GSK said.

At the end of May, Pfizer gained FDA approval of its RSV vaccine for adults 60 and older. Moderna also has conducted a successful phase 3 trial of its RSV shot but has yet to apply for approval. Story.

UPDATED: Tuesday, June 6 at 10:14 a.m. ET

The FDA has converted its accelerated approval for GSK’s Jemperli to a full approval. The treatment is for patients with mismatch repair-deficient (dMMR) recurrent or advanced endometrial cancer, as determined by an FDA-approved test.

Jemperli is for those who have progressed on or following a prior platinum-containing regimen and are not candidates for curative surgery or radiation. The FDA granted the accelerated approval in April of 2021.

The sanction is based on long-term outcomes from a phase 1 trial which showed that 45% of patients responded to treatment, with 15.6% achieving complete response. The median duration of response was 28 months.

Endometrial cancer is found in the inner lining of the uterus and is the most common gynaecologic cancer globally, with approximately 417,000 new cases reported each year, GSK said.
The FDA has signed off on a new indication for Merck’s Prevymis (letermovir), adding it as a preventative measure for cytomegalovirus (CMV) disease in high-risk adult kidney transplant recipients.

In 2017, the U.S. regulator approved Prevymis, a once-daily oral tablet, for prevention of CMV infection in CMV-seropositive recipients of an allogenic hematopoietic stem cell transplant (HSCT).

This new indication was supported by a phase 3 trial of 589 kidney transplant patients with a high risk of CMV infection, which demonstrated that Prevymis is effective and non-inferior to the current standard of care, valganciclovir.
The European Union has granted marketing authorization to Fennec Pharmaceuticals’ Pedmarqsi (sodium thiosulphate) to reduce the risk of hearing loss in oncology patients aged one month to 18 years.

The endorsement for the treatment, known as Pedmark in the United States, comes on top of an FDA approval for it in September of last year. It is the first therapy for the condition, which can accompany cisplatin chemotherapy in patients with localized, non-metastatic solid tumors.

The nod was based on efficacy and safety data from two phase 3 trials which showed that use of cisplatin plus Pedmarqsi reduced hearing loss compared to those who received cisplatin alone.

Permanent bilateral hearing loss occurs in roughly 60 percent of children treated with cisplatin and is often dependent on the dosage and duration of the chemotherapy treatment. There are approximately 10,000 children each year in the U.S. and Europe who take cisplatin chemo, Fennec said.

UPDATED: Monday, May 30 at 8:42 a.m. ET

After an FDA rejection last March for Akebia's vadadustat, the company says the agency has provided it with a "path forward" for the medicine in chronic kidney disease-related anemia for dialysis-dependent patients. While the FDA denied the company's formal appeal of the regulatory rejection, the agency did provide steps for the company to follow to resubmit the drug.

In a letter to Akebia, the FDA outlined flaws with the company's application, including concerns over the risk of thromboembolic events, the company said in a release. The good news is, Akebia doesn't have to run a separate clinical trial.

With the new input, Akebia plans to request a "Type A" meeting with the agency to "ensure alignment on the contents of the ... resubmission." In a statement, Akebia CEO John P. Butler said the company is "extremely pleased with this outcome."

Vadadustat is approved in Japan, where Akebia has a partnership with Mitsubishi Tanabe Pharma Corporation. It's a member of a new class of oral anemia drugs that weathered several regulatory setbacks in the United States.
While Amylyx's amyotrophic lateral sclerosis drug Relyvrio already carries an approval in the United States, the company has now flagged concerns about recent developments in Europe.

After this month's meeting of the Committee for Medicinal Products for Human Use, the group is "trending toward a negative opinion" on the company's application, according to Amylyx. The CHMP is a committee of the European Medicines Agency.

“We disagree with the current view expressed by the CHMP and remain confident in the data from the CENTAUR trial," Amylyx's global head of regulatory affairs and clinical compliance, Tammy Sarnelli, said in a statement. "Should a negative opinion ultimately be issued, we intend to request a formal re-examination procedure."

The re-examination process would take about four months, Amylyx said in a statement.

Meanwhile in the United States, the drug won a groundbreaking FDA approval last September. It's also approved in Canada.

UPDATED: Friday, May 26 at 12:05 p.m. ET

Takeda's Hutchmed-partnered fruquintinib has earned an FDA priority review in previously treated colorectal cancer, with a decision now expected by Nov. 30.

Takeda gained ex-China rights to the VEGFR inhibitor in January by paying Hutchmed $400 million upfront. The drug was approved in China in 2018 under the brand name Elunate based on positive overall survival data from the phase 3 FRESCO trial conducted in China. It later repeated the success in the global FRESCO-2 trial.
Bristol Myers Squibb's Opdivo scored backing from drug reviewers at the European Medicines Agency for an approval as a presurgical neoajudvant treatment for resectable non-small cell lung cancer at high risk of recurrence.

But compared with a broad, all-comers label in the U.S., the EU recommendation only covers patients whose tumors express PD-L1. As BMS noted, Opdivo has been approved in neoadjuvant NSCLC regardless of PD-L1 expression levels across 21 countries.
Roche has voluntarily withdrawn its application for Susvimo for age-related macular degeneration in Europe. The therapy is a drug-device formulation of Lucentis.

Roche made the decision because it wouldn't be able to secure CE certificates for the device before the drug application deadline, a Roche spokesperson told Fierce Pharma. The Swiss pharma plans to resubmit the application to the EMA in the future once it receives the CE certificates, the spokesperson added.

Roche in October 2022 issued a voluntary recall of the Susvimo ocular implant, insertion tool and initial fill kit in the U.S. because of a manufacturing problem. The spokesperson said Roche has made "significant progress" to resolving the issue.
UPDATED: Lundbeck has also pulled an EMA application for Otsuka-partnered Abilify Asimtufii as a maintenance treatment of schizophrenia. The long-acting version of Abilify can be given every two months. And it just earned an FDA approval a month ago.

Lundbeck pulled the application because of a procedural objection from the CHMP, a company spokesperson told Fierce Pharma. Lundbeck plans to resubmit the application as woon as possible under the “line-extension” procedure, rather than the original “hybrid” procedure, the spokesperson said. The company previously announced the decision earlier in May.

UPDATED: Thursday, May 25 at 1:20 p.m. ET

Pfizer's Paxlovid has secured a full approval from the FDA after its emergency use authorization in late 2021.

Specifically, the agency approved the oral antiviral to treat mild-to-moderate COVID-19 in adults who are at a high risk of progression to severe disease. The approval will allow Pfizer to sell the drug commercially when the U.S. government's supplies run out, Reuters reports.

Paxlovid has been a major moneymaker for Pfizer, pulling in nearly $19 billion worldwide last year.
Elsewhere, Celltrion on Wednesday said its Humira biosimilar Yuflyma has picked up an FDA approval. The product is set to launch in July.

Celltrion says its biosimilar version is a high-concentration version of AbbVie's Humira and is citrate-free. On the market, it'll compete with several other biosimilars, including products from Amgen, Pfizer and many other companies.

The company is also working on an interchangeability tag and expects to land that designation in late 2024.

UPDATED: Thursday, May 18 at 9:10 a.m. ET

The National Institute for Health and Care and Excellence (NICE) has blessed AbbVie’s Rinvoq (upadacitinib) for the treatment of moderately to severe Crohn’s disease. The nod comes a year after the JAK inhibitor won approval in the U.K. for ulcerative colitis (UC).

NICE recommends Rinvoq for those who don't respond to biological treatments. The endorsement will open use of Rinvoq to 155,000 people in the U.K. with Crohn’s disease.

Rinvoq has been approved in the U.S. for several indications, hitting the market in 2019 for rheumatoid arthritis. The FDA endorsed the treatment for UC last year but has yet to sign off on Rinvoq’s use for Crohn’s.
NICE also has recommended CSL Vifor and Cara Therapeutics’ Kapruvia (difelikefalin) for the severe itching that accompanies chronic kidney disease (CKD). Kapruvia was approved in the U.K. and Europe a year ago for CKD pruritis.

This nod allows patients to use the three-times-a-week infused treatment as an add-on if other therapies such as creams and antihistamines are ineffective. Pruritis is a common side effect of the dialysis that CKD patients undergo.
Meanwhile, Bristol Myers Squibb’s Sotyktu has received marketing authorization in the U.K. for moderate to severe plaque psoriasis patients who are eligible for systemic treatment. The TYK2 inhibitor and once-daily oral drug was approved by the FDA in September of last year and in Europe in March.

The nods were based on two phase 3 trials that showed Sotyktu achieved skin clearance and improved measures of quality of life. There are few other oral options to treat psoriasis, which affects 1.8 million in the U.K.

UPDATED: Wednesday, May 17 at 10:15 a.m. ET

After a high-profile rejection for Innovent Biologics’ Tyvyt, another China-made PD-1 inhibitor is starting a bid to win an FDA approval.

Elevar Therapeutics has submitted to the FDA an application for a combination of Jiangsu Hengrui Pharma’s PD-1 inhibitor camrelizumab and VEGFR inhibitor rivoceranib, also known as apatinib, in newly diagnosed unresectable liver cancer, the company said Wednesday.

Both drugs have been approved in China, including in the first-line liver cancer indication in February. But neither are approved in the U.S.

If approved, Elevar and Hengrui will go after Roche’s PD-L1/VEGF pairing of Tecentriq and Avastin, which is currently the only FDA-approved immunotherapy for front-line liver cancer.

Elevar’s application is mainly based on data from the phase 3 CARES 310 study. The PD-1/TKI combo significantly slashed the risk of death by 38% over Bayer’s old standard Nexavar. Patients who took the combo lived a median 22.1 months, versus 15.2 months for the Nexavar group.

The filing faces a couple important challenges. First, for any combo therapy, the FDA requires proof of a meaningful contribution from each component. Given that neither camrelizumab nor rivoceranib are available in the U.S., no FDA-approved evidence exists to back their power.

But during an interview with Fierce Pharma back in October, then-Elevar Chief Medical Officer Jan Van Tornout pointed out the two drugs have single-agent data in advanced liver cancer in Chinese patients.

Another problem with CARES 310 is its high representation of Asian patients. The study enrolled 17.3% non-Asian patients, below the FDA’s desire of 20%, because of COVID disruptions, according to Van Tornout.

It’s a bold move for Elevar and Hengrui to target a relatively large cancer indication for a PD-1 inhibitor’s first approval in the U.S. Two other China-made PD-1s—tislelizumab by BeiGene and Novartis and toripalimab by Junshi and Coherus—are initially angling for niche indications and are both awaiting long-delayed FDA decisions.

U.S. FDA NICE European Commission Clinical Data