Regulatory tracker: J&J seeks pediatric FDA approvals for Tremfya

Welcome to Fierce Pharma's regulatory tracker for the second half of 2024.

On this page, we're recording the regulatory progress of in-market products, including expansions into key geographies and new indications.

UPDATED: Monday, Dec. 2 at 8:45 a.m.

• As Johnson & Johnson works to extend the reach of immunology medicine Tremfya, the company has submitted two new supplemental biologics license applications to the FDA.

  The drugmaker seeks approval for Tremfya to treat children ages 6 years and older with moderate-to-severe plaque psoriasis (PsO) and children 5 and older with active juvenile psoriatic arthritis (jPsA).

  In PsO, J&J's filing is based on a phase 3 study called PROTOSTAR, which enrolled pediatric patients with moderate to severe plaque PsO, as well as bridging pharmacokinetic data from a pair of late-stage trials in adults. 

  In jPsA, the filing is based on pharmacokinetic extrapolation analyses from adult studies, as well as efficacy and safety data from the pediatric PROTOSTAR trial.

  "There is a critical gap in the treatment of children and adolescents with these skin and joint conditions, where debilitating symptoms can present challenges related to physical appearance and ability to function," J&J's immunodermatology area disease leader, Liza O'Dowd, M.D., said in a statement. "At Johnson & Johnson, we are working to address this gap by investigating the efficacy and well-characterized safety profile of Tremfya for pediatric patients."

• Biocon Biologics has secured FDA approval for Yesintek, its biosimilar version of J&J's immunology blockbuster Stelara.

  The biosimilar is approved to treat Crohn's disease, ulcerative colitis, plaque psoriasis and psoriatic arthritis, Biocon Biologics said in a press release.

  Based on a prior settlement and licensing agreement, Biocon Biologics has obtained rights to launch its biosimilar in the U.S. no later than February 25.

  Several other Stelara biosmilars have gained FDA approvals, and Amgen is set to launch the first copycat in less than a month.

UPDATED: Wednesday, Nov. 27 at 9:00 a.m.

• In Europe, Novartis' Kisqali has secured approval, when used in combination with an aromatase inhibitor, for the adjuvant treatment of patients with HR-positive, HER2-negative early breast cancer that's at a high risk of recurrence.

  The approval is based on results from the phase 3 NATALEE study, which enrolled a broad patient population with HR+/HER2- stage II and III early breast cancer, Novartis said in a press release.

  The trial showed a "significant and clinically meaningful" 25% reduction in the risk of disease recurrence for adjuvant Kisqali plus endocrine therapy (ET) versus ET alone, Novartis explained.

  The EU approval comes shortly after Kisqali picked up a similar nod in the United States.

• BeiGene's Tevimbra (tislelizumab) has secured approval from the European Commission for the first-line treatment of esophageal squamous cell carcinoma (ESCC) and gastric or gastroesophageal junction (G/GEJ) adenocarcinoma when used in combinations with chemo.

  In ESCC, the approval covers Tevimbra and platinum-based chemo for first-line patients with unresectable, locally advanced or metastatic cancer whose tumors express PD-L1 with a tumor area positivity (TAP) score of 5% or greater, BeiGene said in a release.

  And in G/GEJ, the approval covers Tevimbra's pairing with platinum- and fluoropyrimidine-based chemo for adults with HER2-negative locally advanced unresectable or metastatic cancer whose tumors express PD-L1 with a TAP score of 5% or greater.

  BeiGene leveraged data from two studies, RATIONALE-305 and RATIONALE-306, for the filings.

  “The compelling data from the RATIONALE-305 and 306 trials underscore the unique clinical profile of tislelizumab and its potential to deliver meaningful improvements in outcomes for eligible patients, offering new hope where it’s needed most," Florian Lordick, director and professor of oncology at the University Cancer Center Leipzig, Germany, said in a release.

• GSK's meningococcal disease vaccine Menveo has secured a new approval from the European Commission in a fully liquid presentation, the company said in a Wednesday press release.

  The new vaccine format is approved for use in people 2 and older and does not require reconstitution, GSK said.

  Trials have showed that the fully liquid MenACWY vaccine has similar immunogenicity, tolerability and safety to the existing lyophilised/liquid presentation, GSK noted. The original Menveo approval is unaffected by the new nod.

• Valneva is seeking a FDA label expansion for its chikungunya vaccine, Ixchiq, to potentially include adolescent use in the drug's eligible population.

  The world-first chikungunya vaccine is approved in adults, but Valneva is seeking approval for use in people 12 to 17, the company said in a release.

  In addition, the company submitted new data to the regulator showing the vaccine’s immune response sustained over 24 months in nearly all participants in a long-term trial.

UPDATED: Monday, Nov. 25 at 12:25 p.m.

• The FDA has accepted GSK's regulatory filing for two important Blenrep combinations, the company said in a Monday release.

  In one combination, the company seeks an approval for Blenrep, Takeda's Velcade and dexamethasone (BVd) in patients with multiple myeloma who have received at least one prior line of therapy. The company also seeks approval for Blenrep's combo with Bristol Myers Squibb's Pomalyst and dexamethasone (BPd) in the same patient group.

  The FDA assigned the filing an approval decision date of July 23, 2025, GSK said. In its press release, the company said the combinations could "redefine multiple myeloma treatment at or after first relapse."

  For its filing, GSK is leveraging data from the DREAMM-7 and DREAMM-8 studies. The trials showed "statistically significant efficacy, including overall survival" in the DREAMM-7 head-to-head study against Johnson & Johnson's myeloma stalwart Darzalex.

  In late 2022, GSK pulled Blenrep from global markets after a study failed to prove its benefit as a monotherapy. But GSK's efforts to revive the drug have yielded success and the company now sees the drug eventually reaching blockbuster heights.

• In the acceptance of another application, the FDA has agreed to review Alynlam’s Amvuttra (vutrisiran) as a treatment for ATTR amyloidosis with cardiomyopathy (ATTR-CM). With the company using a priority review voucher, the FDA has set an action date of March 23 to decide whether to approve Amvuttra in the indication. The agency does not plan to hold an advisory committee meeting to review the application.

  With an approval to treat ATTR-CM, Alnylam would challenge Pfizer’s Vyndaqel/Vyndamax franchise, which dominates the indication, generating sales of $3.9 billion in the first three quarters of this year. Also in the mix is BridgeBio’s Attruby, a potential blockbuster approved by the U.S. regulator on Friday. 

  As a TTR gene silencer, Amvutta would bring a drug with a new mechanism of action to treat ATTR-CM. 

  In June of 2022, the FDA signed off on Amvuttra in another indication—polyneuropathy of hereditary transthyretin—which has a smaller patient population than ATTR-CM. In the first three quarters of this year, Amvuttra has racked up $685 million in sales.

UPDATED: Friday, Nov. 22 at 10:10 a.m.

• Johnson & Johnson is looking to follow up on its recent Tremfya FDA approval in ulcerative colitis with a nod for the subcutaneous form of the drug in the same indication.

  The company submitted its FDA bid for the subcutaneous regimen based on the phase 3 ASTRO study, which showed that a 400 mg dose of Tremfya, administered subcutaneously at weeks 0, 4 and 8, delivered statistically significant and clinically meaningful remission results at week 12. 

  If approved, the drug would be the only FDA-approved IL-23 inhibitor that can be used intravenously or subcutaneously for ulcerative colitis patients, according to J&J. 

  In addition, J&J has submitted filings for Tremfya as a Crohn’s disease treatment in the U.S. and Europe. 

• GSK’s respiratory syncytial virus (RSV) vaccine Arexvy can now offer protection to a wider group of adults in Japan.

  The country’s Ministry of Health, Labour and Welfare expanded the shot’s pool of eligible recipients to people ages 50 to 59 who are at increased risk of RSV. 

  Before the nod, adults 60 and older were eligible to receive the vaccine in the country after the regulatory agency greenlit the shot in September of 2023.

• Merck’s Welireg is the first and only systemic therapy to win approval in China for adult patients with certain von Hippel-Lindau (VHL) disease-associated tumors, the company said.

  China’s National Medical Products Administration cleared the drug for adult VHL disease patients who require therapy for associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas or pancreatic neuroendocrine tumors (pNET), not requiring immediate surgery, according to a Merck release.

  Until now, that patient population hadn’t been able to access to a non-surgical treatment option. 

  Welireg is the first hypoxia-inducible factor-2 alpha inhibitor to hit the market in China. Merck is also studying the treatment in advanced RCC and other tumor types as a monotherapy and combined with other medicines. 

UPDATED: Thursday, Nov. 21 at 9:35 a.m.

• The European Commission has approved Pfizer's Hympavzi for patients 12 and older who weigh at least 35 kg (77 pounds) with severe hemophilia A or B.

  Specifically, the regulatory endorsement covers patients with severe hemophilia A without FVIII inhibitors and those with severe hemophilia B without FIX inhibitors. 

  The drug is the the first and only anti-tissue factor pathway inhibitor to be approved in Europe and the first hemophilia drug approved in the region given via a pre-filled, auto-injector pen, Pfizer said in a release. The European nod comes about a month after the FDA signed off on the drug.

  “This approval builds on Pfizer’s more than four-decade commitment to improve the standard of care in hemophilia, and we look forward to delivering this medicine that reduced bleeds as compared to factor prophylaxis and, importantly, requires limited preparation, meeting a key need for eligible patients," Pfizer's chief international commercial officer, Alexandre de Germay, said in a statement this week.

• In the U.S., the FDA has signed off on a label change for a decades-old cancer drug. 

  The drug, Sandoz's fludarabine phosphate, is now approved as part of a combination to treat adults with B-cell chronic lymphocytic leukemia (CLL), according to the agency. The FDA also endorsed the drug for adults with B-cell CLL who have not responded to or whose disease has progressed during treatment with at least one alkylating-agent containing regimen.

  The agency further adjusted its dosing recommendation for the drug when it's used in combination with cyclophosphamide and rituximab.

  The changes came as part of Project Renewal, which seeks to update labels for older cancer medicines. Fludarabine phosphate was first approved in 1991.

• A Bayer supplemental new drug application (sNDA) for Nubeqa has been accepted by the FDA. 

  The company seeks an approval for the drug, used in combination with androgen deprivation therapy (ADT), for the treatment of patients with metastatic hormone-sensitive prostate cancer (mHSPC), according to Bayer's release.

  Currently, Nubeqa is approved to treat adults with mHSPC in combination with docetaxel and to treat adults with non-metastatic castration-resistant prostate cancer.

  The current sNDA leverages results from Bayer's phase 3 ARANOTE study. The trial showed that the combo significantly extended the time before tumor progression or death compared with ADT alone. 

UPDATED: Tuesday, Nov. 19 at 8:45 a.m.

• The FDA has issued a complete response letter to Astellas after the company filed a supplemental biologics license application for its geographic atrophy drug Izervay.

  Astellas had sought to incorporate positive 2-year data from a study called GATHER2 in the drug's label. The GATHER2 study evaluated monthly and every-other-month (EOM) dosing for the drug in GA patients through 2 years.

  Rather than flagging concerns over safety or the drug's risk/benefit profile, the FDA raised "a statistical matter related to labelling language proposed by Astellas," according to the company's Monday press release.

  In a note to clients, Mizuho analyst Graig Suvannavejh noted that the development could represent an "incremental positive" for Astellas' rival in the space, Apellis, because the latter company's drug, Syfovre, already has monthly and EOM dosing on its label. Izervay's label only describes monthly dosing. 

  While Astellas works out its issue at the FDA, this dynamic allows "Syfovre to enjoy somewhat of a competitive advantage over Izervay (at least for the time being)," Suvannavejh wrote.

• Meanwhile, Novo Nordisk has launched its popular weight loss drug, Wegovy, in China, according to press reports.

  Wegovy carries a monthly list price of around $193 in China, Bloomberg reports, citing local press, which is just a fraction of the drug's $1,349 monthly list price in the U.S.

  Many patients in China will have to pay out of pocket to access the drug, according to Bloomberg. 

UPDATED: Friday, Nov. 15 at 9:30 a.m.

• A new indication for Sanofi and Regeneron’s blockbuster Dupixent could be coming in April of 2025 after the FDA accepted the partners' resubmitted supplemental biologics license application for the drug in chronic spontaneous urticaria (CSU).

  If approved, Dupixent would be the first new targeted therapy for the chronic inflammatory skin disease in a decade. In three trials within the LIBERTY-CUPID phase 3 program, the drug significantly reduced itch and hives, two hallmarks of the disease.

  A CSU nod could open Dupixent up to the more than 300,000 people in the U.S. who have CSU that isn’t adequately controlled by antihistamines. Dupixent already has a CSU indication in Japan and in the United Arab Emirates. 

• Sandoz’ biosimilar to Regeneron and Bayer’s Eylea was approved in Europe as Afqlir, paving the way for a late-2025 launch.

  The European Commission granted marketing authorization for the med’s use in several retinal diseases, including neovascular age-related macular degeneration. Throughout France, Germany, Italy, Spain, the U.K., the U.S. and Japan, an estimated 3.6 million patients have the vision-impairing disease.

  Afqlir is one of several of Sandoz’s key biosimilars and was approved in August by the FDA under the brand name Enzeevu. 

• Novavax’s newest version of its Nuvaxovid COVID-19 vaccine was approved in the U.K., offering protection from the latest Omicron JN.1 COVID subvariant.

  The Medicines and Healthcare products Regulatory Agency (MHRA) approved the adapted vaccine under the International Recognition Procedure, a regulatory pathway that allows the MHRA to speed up an evaluation using the decision-making of other drug reviewers around the world.

  Novavax’s latest Nuvaxavoid version was cleared for use as Nuvaxovid JN.1 for those aged 12 years old and older. 

UPDATED: Wednesday, Nov. 13 at 9:40 a.m.

• After England's National Institute for Health and Care Excellence (NICE) turned down the combination of Sanofi's Sarclisa with pomalidomide and dexamethasone for certain patients with multiple myeloma, the French drugmaker celebrated progress in overturning the agency's recommendation.

  NICE will convene a third committee meeting to discuss the combination, known as Isa-Pd, in fourth-line patients with relapsed or refractory multiple myeloma, the company said in a Tuesday release. 

  The decision follows a "successful appeal" of NICE's final draft guidance published in June, Sanofi said, noting that several of its appeal points were acknowledged by the agency. For instance, under NICE's "standard methodology," the combination "cannot be deemed cost effective" even if Sanofi provides the Sarclisa component for free, according the drugmaker. 

  The specific fourth-line indication under review is for patients who've tried lenalidomide and a proteasome inhibitor, and whose disease has progressed on their most recent treatment.

  Through its use on England's Cancer Drugs Fund, the Isa-Pd combo has become a standard-of-care regimen for eligible patients, Sanofi said in its release. Isa-Pd will continue to be available on CDF while NICE's evaluation is underway.

UPDATED: Tuesday, Nov. 5 at 8:10 a.m.

• Organon has disclosed a 3-month regulatory delay for a Vtama label expansion bid shortly after closing its acquisition of dermatology specialist Dermavant.

  The FDA's review process for Vtama in patients with atopic dermatitis ages 2 and older will take 3 months longer than expected, Organon said Tuesday. The agency is now expected to make its decision by March 12, 2025. 

  Rather than spotlighting any concerns with safety or efficacy, the FDA determined that it would need more time to review final data from a long-term extension study of the drug, Organon said.

  With the delay, Organon expects Vtama to bring in $125 million next year. That's a slight reduction from a $150 million projection issued last week.

  Organon acquired the drug through its recent Dermavant purchase for up to $1.2 billion. Vtama is already approved as a topical treatment for plaque psoriasis in adults.

UPDATED: Friday, Nov. 1 at 9:55 a.m.

• Eisai has completed its rolling FDA submission for a weekly subcutaneous maintenance dose of Alzheimer's disease therapy Leqembi, the company said in a Friday press release.

  The company's filing is based on data from an open-label extension phase of its Clarity AD study, plus "modeling of observed data," the drugmaker said in the release. 

  If approved, the subcutaneous autoinjector would be used to administer patients' maintenance doses at home or in medical facilities, potentially alleviating some of the infrastructure hurdles currently affecting the Leqembi rollout. As it stands, the drug's maintenance doses are given during one-hour infusions every two weeks. The company is also seeking FDA approval for a monthly IV maintenance dose.

• Even as PTC Therapeutics faces a potential Translarna market withdrawal in Europe, the company is pursuing an approval for its Duchenne muscular dystrophy medicine in the United States.

  The FDA has accepted PTC's resubmission for Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD), the company said in a Wednesday release. The regulatory development comes many years after the agency rejected a prior PTC approval bid back in 2017.

  For its new filing, the company submitted data from a placebo-controlled trial called Study 041. According to PTC, the drug showed "significant benefit" on the trial endpoints of six-minute walk distance, 10-meter walk/run test and a measure called the NorthStar Ambulatory Assessment.

  Regulators in Europe last month recommended the drug be pulled from the market. At the time of the decision, PTC expected the European Commission to make its final call on pulling the drug in about 67 days.

UPDATED: Tuesday, Oct. 29 10:30 a.m.

• England's National Institute for Health and Care Excellence (NICE) has recommended Pfizer's Elrexfio for patients with multiple myeloma who've tried at least three other kinds of treatments and whose cancer has worsened after their most recent treatment.

  Under the recommendation, the drug is set to be available on England's Cancer Drugs Fund as officials continue to collect more data on its use, according to a NICE release.

  Previously, NICE had signed off on the drug after three or more lines of treatment and only if pomalidomide plus dexamethasone would have otherwise been offered to patients. NICE's reviewers removed the restriction on the drug based on "further analysis" provided by Pfizer, according to the release.

• Besides the Pfizer endorsement, AstraZeneca gained a NICE nod for Wainzua to treat hereditary transthyretin-related amyloidosis in adults with stage 1 or stage 2 polyneuropathy.

  The drug, which can be taken at home, recently won an approval from the Medicines and Healthcare products Regulatory Agency (MHRA) in the country. 

  NICE estimated that 12 people could benefit from the new therapy during the first year after the recommendation.

• In the U.S., Shorla's Oncology's Jylamvo gained FDA label expansions to treat pediatric patients with acute lymphoblastic leukemia (ALL) and polyarticular juvenile idiopathic arthritis (pJIA).

  The approval makes Shorla's medicine the only oral liquid methotrexate approved by the FDA for adults and pediatric uses, according to a release from the company.

  Previously, the medicine had secured FDA approvals to treat adults with ALL, mycosis fungoides, relapsed or refractory non-Hodgkin lymphoma, rheumatoid arthritis and severe psoriasis, according to Shorla.

UPDATED: Monday, Oct. 28 8:55 a.m.

• Eli Lilly's Mounjaro (tirzepatide) has picked up approvals to help with weight management and for patients with insufficiently controlled diabetes in Hong Kong. 

  In weight management, the drug is approved to treat adults with obesity—or those who are overweight—who have associated medical complications such as hypertension or cardiovascular disease, according to an Eli Lilly press release.

  In diabetes, the drug is approved as an adjunct to diet and exercise to improve glycemic control in diabetic patients when metformin is "considered inappropriate," or in addition to other medicines to treat the disease.

  The company is planning to launch the drug in Hong Kong by the end of the year, according to a Bloomberg News report.

  While Lilly's drug carries similar approvals in China, it's unclear when Lilly plans to launch Mounjaro in the country, according to Bloomberg.

UPDATED: Thursday, Oct. 24 at 1:25 p.m.

• Merck’s Keytruda collected two new indications in Europe, bringing its total number of approved uses in the region up to 30.

  The oncology superstar won approval as a first-line treatment for primary advanced or recurrent endometrial carcinoma in adult patients who are candidates for systemic therapy when used in combination with the chemotherapies carboplatin and paclitaxel.

  Keytruda also picked up a separate indication for FIGO stage 3/4a locally advanced cervical cancer in adults who have not received prior definitive therapy, alongside chemotherapy.

  Keytruda, which has 41 indications in the U.S., can now claim 30 nods in the EU, including five in gynecologic cancers. 

• AstraZeneca’s Voydeya picked up an endorsement from England’s National Institute for Health and Care Excellence (NICE) as an add-on treatment option for certain patients with paroxysmal nocturnal hemoglobinuria (PNH).

  Voydeya is a first-in-class oral factor D inhibitor designed to be added on to AZ’s Ultomiris or Soliris in PNH patients who have residual hemolytic anemia and clinically significant extravascular hemolysis while on a C5 inhibitor. NICE recommended the drug based on a phase 3 study in which Voydeya was associated with a meaningful change in hemoglobin levels from baseline to week 12.

  So far, the med has been approved in the U.S., the U.K., Europe and Japan. The company is also weighing its use as a monotherapy across other indications. 

UPDATED: Monday, Oct. 21 at 9:20 a.m.

• After the FDA rejected the combination of the PD-1 inhibitor camrelizumab and the VEGFR tyrosine kinase inhibitor rivoceranib earlier this year, the agency will take another look at the proposed first-line liver cancer regimen.

  The FDA has accepted a resubmission filing for the combination as a potential first-line systemic treatment for unresectable or metastatic hepatocellular carcinoma (uHCC), Elevar Therapeutics said Monday.

  In the final analysis of the CARES-310 study, the combination was associated with median overall (mOS) survival of 23.8 months in uHCC patients who hadn't received prior systemic treatments. That result is the "longest mOS for any treatment in a global Phase 3 trial for patients with uHCC," according to Elevar's Monday release.

  The agency has assigned the filing a target decision date of March 20, 2025. The length of the review isn't the best case Elevar had hoped for.

  Previously, the FDA rejected the proposed combination because of findings it identified during a manufacturing inspection for camrelizumab.

  Both drugs are approved in China. Early last year, the pairing won approval in China as a first-line treatment for uHCC.

• Amneal has received FDA approval for pyridostigmine bromide extended-release tablets as a pretreatment for soman nerve agent poisoning. The drug has been developed for the U.S. armed forces and U.S. allies with support from the government, Amneal said in a Monday release.

  The product was not previously disclosed in Amneal's pipeline.

  "This new pyridostigmine therapy speaks to Amneal’s expertise in developing new innovative product formulations and our ongoing work with the U.S. government on new solutions," Amneal's co-CEOs Chirag and Chintu Patel said in a joint statement. "With the approval of pyridostigmine bromide extended-release tablets, we are proudly supporting the U.S. military."

UPDATED: Friday, Oct. 18 at 8:50 a.m.

• The FDA will take another three months to decide whether to approve Amgen's application for Lumakras in combination with Vectibix in patients with chemorefractory KRAS G12C–mutated metastatic colorectal cancer.

  The agency originally assigned the filing target decision date of October 17, but it now aims to decide by January 17, 2025, an Amgen spokesperson said in an email. The delay will "allow additional time for review of supplemental data recently submitted to the agency," the spokesperson said.

  A year ago, phase 3 trial results showed that the combination delivered statistically significant improvements in progression-free survival compared to an investigator's treatment of choice. At a median follow-up of 7.8 months, PFS reached 5.6 months for the Lumakras 960 mg group and 3.9 months for the 240 mg group, compared with 2.2 months for the control group.

• Even as Eisai and Biogen work to accelerate the launch of Alzheimer's drug Leqembi in the U.S. and elsewhere, the global rollout has hit a snag in Australia.

  In an interim decision, the country's Therapeutic Goods Administration has decided not to approve the medicine as a treatment for patients with mild cognitive impairment due to Alzheimer’s disease (AD) and mild AD dementia, Eisai said in a release.

  Eisai plans to request a reconsideration of the decision, the company added.

  The drug is approved in the U.S., Japan, China, South Korea and several other countries. In another setback for the therapy, regulators in Europe rejected Leqembi this past summer.

UPDATED: Wednesday, Oct. 16 at 4:00 p.m

• England’s National Institute for Health and Care Excellence (NICE) gave its first endorsement to a targeted post-surgery treatment option for ALK-positive non-small-cell lung cancer (NSCLC) to Roche’s Alecensa.

  Just under 100 people in England are eligible for the treatment, which is given in twice daily tablets to completely remove stage 1B tumors after surgery. The drug works by targeting cancer cells with rearranges in the ALK gene.

  The FDA previously approved the therapy in that indication earlier this year, a month ahead of schedule, based on studies showing that Alecensa cut the risk of disease recurrence or death by 76% compared with platinum-based chemotherapy.   

• NICE also recommended Merck's Keytruda’s use before surgery with chemotherapy and on its own after surgery to treat resectable NSCLC.

  That option has a patient population of over 5,000 eligible patients in England. In studies, Keytruda paired with platinum-based chemotherapy before surgery and then as a monotherapy after surgery decreased the potential for events that would prevent surgery or for the cancer to return following surgery compared with placebo, as well as increased overall survival rates.

  Keytruda took home its first approval in that indication from the FDA last October. Bristol Myers Squibb’s rival Opdivo picked up the same FDA nod earlier this month. 

• Patients in the U.K. with polyneuropathy associated with rare disease hereditary transthyretin-mediated amyloidosis (ATTRv-PN) will soon have a new treatment option in AstraZeneca and Ionis Pharmaceuticals’ Wainua.

  The U.K.’s Medicines and Healthcare products Regulatory Agency approved the drug in a pre-filled pen to treat the polyneuropathy symptom of ATTRv, which results in pain, mobility difficulties and weakness due to damage to multiple nerves.

  Wainua works by lowering the amount of transthyretin protein made by the liver. In ATTRv patients, the protein clumps together to form amyloid deposits, which can build up around or within the nerves, heart and other organs and prevent them from working properly. In a 168-patient-wide study, those treated with Wainua had a greater reduction in transthyretin protein levels and less disease progression from baseline than those in the placebo arm. 

• The FDA updated the label for Sanofi’s Flublok influenza vaccine to include data from a large safety study in pregnant people.

  The post-licensure, observative, retrospective safety surveillance study, included more than 48,700 pregnant participants over multiple flu seasons. In the study, Sanofi found that its vaccine’s safety profile in pregnant people is consistent with pregnancy outcomes in the general population. No increased risk of birth or neonatal outcomes were observed over the study.

  The updated label comes before the U.S. Centers for Disease Control and Prevention (CDC) recommendations to receive a flu vaccine before the end of October. 

• GSK‘s bid to bring a new urinary tract infection (UTI) med to market was accepted by the FDA with a decision date in March 2025.

  If approved, the company’s gepotidacin would be the first in a new class of oral antibiotics for uncomplicated UTIs in over 20 years. New treatments for the common infection are crucial as UTIs caused by drug-resistant bacteria are on the rise and can lead to higher treatment failure rates, according to GSK.

  In phase 3 studies, the candidate proved non-inferiority to standard of care nitrofurantoin in female adults and adolescents with a confirmed UTI and achieved therapeutic success in more patients than in the nitrofurantoin arm. Gepotidacin was also successful in treating gonorrhea in a separate trial and is designed to have a lower potential of antibiotic resistance.  

• Valneva’s shigellosis vaccine candidate Shigella4V, which it licensed out from LimmaTech Biologics, was granted a speedy review with the FDA’s fast track designation.

  Shigellosis is the second leading cause of diarrheal deaths worldwide with an estimated 600,000 deaths linked to the bacteria. Treatment is “extremely difficult,” according to Valneva, as the bacterial infection is highly resistant to antibiotics and has evolved into multi-drug-resistant strains. There is so far no licensed vaccine available to prevent infection.

  Velneva paid LimmaTech over $10 million for a global Shigella4V license in August. LimmaTech will initiate a phase 2 controlled human infection model study and a phase 2 pediatric study by the end of this year, after which Valneva will take over all further development and handle commercialization if approved. 

UPDATED: Monday, Oct. 14 at 4:30 p.m.

• Johnson & Johnson’s multiple myeloma med Tecvayli won a broader recommendation from England’s National Institute for Health and Care Excellence (NICE) in its final draft guidance.

  The recommendation applies to U.K. patients with relapsed and refractory multiple myeloma who have received at least three lines of specific therapies and have progressed on their last treatment. Previously, NICE’s endorsement limited Tecvayli’s use to patients who would otherwise receive treatment with a pomalidomide and dexamethasone combo regimen.

  Scotland was the first in the U.K. to approve the treatment in September based on trial data that showed 63% of patients taking Tecvayli had an overall response to the treatment, with 46% meeting the bar for complete response. The FDA and the European Commission both granted approval in 2022. 

• UCB’s Bimzelx has been cleared for use in a new single-injection regimen with a pre-filled syringe and pre-filled autoinjector for its 320mg dose strength.

  The newly FDA approved device options are meant to boost patient experience and convenience for those with moderate-to-severe plaque psoriasis who need a 320mg dose of the drug, the company said in a release. That dosing strength is recommended for Bimzelx’ plaque psoriasis and psoriatic arthritis with coexistent plaque psoriasis indications, while non-radiographic axial spondyloarthritis and ankylosing spondylitis patients use a 160mg dose.

  UCB currently markets a 1 mL administration option that contains 160mg of the drug. The newest 320mg delivery methods were previously approved by the European Commission in August.  

• AstraZeneca and Daiichi Sankyo’s Enhertu picked up a new conditional approval from China’s National Medical Products Administration.

  With the nod, the drug can treat patients with unresectable, locally advanced or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 mutations and who have received a prior systemic therapy. China’s lung cancer patient population makes up more than 40% of global lung cancer patients with more than one million people diagnosed yearly. 

  The approval was based on two phase three studies. In the DESTINY-Lung 02 trial, Enhertu proved a confirmed objective response rate of 49% and a median duration of response of 16.8 months, plus a median progression-free survival of 9.9 months and median overall survival at 19.5 months. Full approval rides on a confirmatory trial, according to AstraZeneca. 

UPDATED: Thursday, Oct. 10 at 11:20 a.m.

• Alnylam has filed Amvuttra with the FDA for an approval in transthyretin amyloid cardiomyopathy (ATTR-CM). To expedite the process, the company has utilized a priority review voucher, which could mean an FDA decision comes by April 2025.

  During an investor event Wednesday, Alnylam's Chief Medical Officer Pushkal Garg, M.D., said the company doesn't expect the FDA will hold an advisory committee meeting to discuss the application because Amvuttra has already shown a benefit on a hard clinical outcomes endpoint in the HELIOS-B trial. 

  As to the potential indication, Garg said Alnylam thinks the HELIOS-B data support broad use of Amvuttra both as a monotherapy and among patients who are on background treatment with Pfizer's tafamidis, which is known commercially as Vyndaqel or Vyndamax.

UPDATED: Wednesday, October 9 at 9:40 a.m.

• The National Institute for Health and Care Excellence (NICE) has rejected Takeda's colorectal cancer drug Fruzaqla for coverage on NHS England. Takeda in-licensed ex-China rights to the VEGFR inhibitor from Hutchmed in early 2023. 

  Pooled data from two trials, FRESCO and FRESCO 2, found that Fruzaqla significantly reduced the risk of death by 34% compared with placebo in patients with previously treated metastatic colorectal cancer. However, reviewers at NICE, using indirect comparison, argued that "there is no difference in how long people live" between Fruzaqla and other existing treatments, according to a draft guidance (PDF).

  The drug cost watchdog therefore concluded that Fruzaqla "was unlikely to represent a cost-effective use of NHS resources." Takeda has a confidential commercial arrangement, but that apparently didn't persuade NICE.

  NICE's decision is not final. The draft guidance is now open for comments, with a second NICE evaluation committee meeting scheduled for mid-December.

• U.K.'s Medicines and Healthcare products Regulatory Agency has approved Ipsen's Iqirvo to treat the rare liver disease primary biliary cholangitis (PBC), following an FDA go-ahead in June. 

  PBC is marked by damages in small bile ducts, which can lead to bile build-up and then liver damage. Iqirvo works by reducing the amount of bile acids the liver produces and reducing the build-up of bile.

  In the ELATIVE trial, 51% of patients receiving Iqirvo achieved a cholestasis response after one year of treatment, compared with 4% of those on placebo. What's more,15% of Iqirvo takers showed normal levels of Alkaline Phosphatase (ALP) and bilirubin, which are markers of liver damage, versus 0% in the placebo arm after one year. 

  Besides Ipsen, several companies are targeting PBC, including Gilead Sciences, Calliditas Therapeutics and Intercept Pharma.

UPDATED: Tuesday, October 8 at 9:30 a.m.

• Biogen's novel therapy for amyotrophic lateral sclerosis (ALS) has secured an approval in China, the company said through its official WeChat account.

  The medicine, which bears the brand name Qalsody in the United States, becomes the first disease-modifying medicine for adult ALS patients in China who have SOD1 gene mutations, the company said.

  Qalsody picked up its U.S. nod last year and launched in Europe earlier this year. The drug, which is still gaining commercial momentum, pulled down $9.6 million during the first half of 2024.

• Also in China, Shijiazhuang-based CSPC Pharmaceutical said that the National Medical Products Administration (NMPA) has approved Enyitan, its biosimilar version of Novartis' Xolair.

  Enyitan is approved to treat patients 12 and older with chronic spontaneous urticaria who've tried antihistamines but remain symptomatic, CSPC said in a release (PDF).

  Head-to-head studies have shown that the product is "highly similar" to the reference medicine in quality, safety and efficacy, CSPC noted. The company is pursuing another indication for the biosimilar, according to the release.

  In China, Xolair is also approved to treat patients with allergic asthma who remain symptomatic after receiving antihistamines.

UPDATED: Thursday, October 3 at 9:10 a.m.

• In AstraZeneca's label expansion bid for Calquence in previously untreated mantle cell lymphoma (MCL), the FDA has awarded a priority review.

  With the priority review designation, AstraZeneca expects the agency to make a decision on its application during the first quarter of 2025. 

  In the phase 3 ECHO trial, the combination of Calquence, Roche's Rituxan and bendamustine reduced patients' risk of disease progression or death by 27% compared to standard-of-care chemo. The addition of Calquence to the standard of care provided almost 1.5 years of additional median progression-free survival, AZ said in a release.

  Calquence is already approved to treat patients with MCL who've received at least one other therapy.

  The Calquence priority review comes shortly after the FDA awarded the same designation for AZ and Daiichi Sankyo's Enhertu in HER2-low or HER2-ultralow breast cancer patients who have received at least one endocrine therapy in the metastatic setting.

• CSL Behring's hemophilia B gene therapy, Hemgenix, has secured a reimbursement recommendation in Spain, the company said in a release.

  The Spanish Interministerial Commission on the Pricing of Medicines' recommendation will result in nationwide reimbursement for eligible patients, the company said. The gene therapy brings a "paradigm shift," offering patients a "durable alternative to frequent intravenous Factor IX infusions, which can significantly impact the quality of life and wellbeing of patients," the company said in its release.

  Hemgenix is approved in the country for adults with severe and moderately severe haemophilia B without a history of Factor IX inhibitors.

UPDATED: Tuesday, October 1 at 10:30 a.m.

• AstraZeneca and Daiichi Sankyo have secured an FDA priority review for Enhertu in HER2-low or HER2-ultralow breast cancer in patients who have received at least one endocrine therapy in the metastatic setting. The FDA action date is February 1, 2025, according to Daiichi (PDF). If approved, Enhertu could move earlier in the treatment of HER2-low breast cancer and reach patients with even lower expressions of HER2.

  Enhertu's current HER2-low indication allows it to treat tumors with a HER2 expression score of at least IHC 1+, whereas HER2-ultralow tumors are defined as IHC 0 with membrane staining. 

  The application is based on results from the phase 3 DESTINY-Breast06 trial. In the study, Enhertu reduced the risk of disease progression or death by 37% by blinded independent central review versus chemotherapy in the overall trial population. In an exploratory analysis, the HER2-directed antibody-drug conjugate numerically reduced that risk by 22% in HER2-ultralow patients.

• Johnson & Johnson has filed an application with the FDA for its subcutaneous Darzalex Faspro in combination with bortezomib, lenalidomide and dexamethasone (VRd) for newly diagnosed multiple myeloma for whom stem cell transplant is either deferred are not appropriate. The filing follows Sanofi's rival approval for its Sarclisa in the same VRd combination in first-line transplant-ineligible myeloma.

  In the phase 3 CEPHEUS trial. Darzalex-VRd significantly reduced the risk of progression or death by 43% when pitted against VRd alone. On the study's primary endpoint, the Darzalex-containing regimen also significant improved the rate of patients who achieved a deep response known as minimal residual disease negativity. MRD-negativity rate as measured by a 1 in 100,000 sensitivity was 60.9% for the Darzalex regimen, versus 39.4% for the control arm at a median follow-up of 58.7 months.

• The FDA has updated an Emergency Use Authorization fact sheet to healthcare providers about Invivyd's COVID-19 prevention med Pemgarda. 

  According to Invivyd, the FDA has removed a line which previous stated that "preliminary, non-peer-reviewed data in the public domain indicate that KP.3.1.1 may have substantially reduced susceptibility to pemivibart." KP.3.1. currently accounts for more than half of circulating variants in the U.S., according to the CDC.

  Based on new in vitro neutralization data, FDA has determined that "Pemgarda is likely to retain adequate neutralization activity against KP.3.1.1," the agency said Sept. 26.

UPDATED: Monday, September 30 at 11:00 a.m.

• The FDA and the European Union have blessed Formycon and Fresenius Kabi’s Otulfi, their biosimilar version of Johnson & Johnson’s Stelara. The nods came hours apart on Friday of last week and cover their intravenous and subcutaneous formulations of the copycat.

  In Europe, Otulfi (ustekinumab-aauz) is approved for the treatment of Crohn’s disease, moderate to severe plaque psoriasis and active psoriatic arthritis. The U.S. nod covers the three indications plus ulcerative colitis.

  Last year, the companies signed an agreement which allows Fresenius to market Otulfi in the U.S. and many other key markets. Germany-based Formycon will retain commercial rights to Otulfi in its home country and in Latin America.

  The companies have reached a settlement with J&J regarding commercialization of the product in Europe and Canada, The agreement remains confidential, Formycon said.

  Other companies that are in line to launch their Stelara biosimilars in the U.S. in early 2025—after signing settlements with J&J—include Biocon Celltrion and Amgen, as well as partners Alvotech and Teva and Samsung Bioepis and Sandoz. 

• The European Commission has signed off on Johnson & Johnson’s combination treatment Yuvanci for patients with pulmonary arterial hypertension (PAH). The single tablet combines J&J’s Opsumit (macitentan) and United Therapeutics' Adcirca (tadalafil), which are commonly prescribed together to treat the condition.

  Yuvanci includes a 10 mg dose of macitentan and a 40 mg dose of tadalafil. The nod applies to patients who are already treated with the combo as separate tablets.

  Backing the endorsement is a phase 3 study which showed that the combo reduced pulmonary vascular resistance by 29% compared to macitentan and 28% for those on tadalafil.   

  In March, the combo was approved in the U.S., where it is marketed as Opsynvi. That nod came the same week that the FDA blessed Merck’s first-in-class PAH drug Winrevair (sotatercept).

• The FDA has converted its accelerated approval for Eli Lilly’s Retevmo (selpercatinib) into a full nod. The endorsement covers patients ages 2 and older with advanced metastatic medullary thyroid cancer (MTC) with a RET mutation, as detected by an FDA approved test.

  The first-in-class treatment was originally blessed on an accelerated basis by the FDA in 2020 for patients age 12 and older. Four months ago, another accelerated nod came through in the indication for patients age 2 and older.

  A year ago, a phase 3 trial of Retevmo paved the way for the full endorsement as it showed that it cut the risk of disease progression or death by 72% versus Exelixis’ Cabometyx or Sanofi’s Caprelsa in patients with MTC who had not previously received a kinase inhibitor.

  In 2022, the FDA gave a thumbs up on an accelerated basis for Retevmo to treat RET solid tumors of any kind.  

UPDATED: Friday, September 27 at 10:45 a.m.

• England’s Medicines and Healthcare Products Regulatory Agency (MHRA) has approved Pharming’s Joenja to treat the rare genetic disease phosphoinositide 3-kinase delta syndrome (APDS), which leaves patients unable to fight infections because their immune system is compromised.

  Joenja—the first disease-modifying treatment for the condition—was approved by the FDA in March of 2023. Patients with APDS have historically been given antibiotics, immunosuppressants and immunoglobulin replacement drugs to deal with the disease symptoms.   

  The oral treatment, which is taken twice daily, is for patients 12 and older who weigh at least 45 kg (99 pounds). Symptoms typically appear in the first two years of life and often include lung infections and stunted growth. Patients with APDS are more prone to develop blood cell cancers such as lymphoma.

  In 2019, the Netherlands-based company spent $20 million to license the investigational therapy from Novartis. In the second quarter, Joenja generated $11.1 million, a 16% increase from the previous quarter. 

• Bayer has submitted a supplemental new drug application to the FDA for Nubeqa to treat metastatic hormone-sensitive prostate cancer (mHSPC) in a combination that does not include chemotherapy. The request is for Nubeqa to be paired with androgen deprivation therapy (ADT).

  In the phase 3 ARANOTE trial, the combination produced a 46% reduction in the risk of disease progression or death compared to ADT alone.

  With an FDA nod, Nubeqa would be approved for the entire mHSPC population. Two years ago, the FDA signed off on a combination of Nubeqa, ADT and the chemotherapy docetaxel. Some patients are not suitable for docetaxel.

  Nubeqa was originally approved in 2019 for non-metastatic, castration-resistant prostate cancer. Nubeqa rolled up sales of 869 million euros ($940 million) in 2023, good for an 86% growth rate.

• China has signed off on Regeneron and Sanofi’s Dupixent as an add-on maintenance treatment for chronic obstructive pulmonary disorder (COPD), joining the FDA which also signed off on Dupixent in the indication on Friday. The nods came two months after Europe endorsed Dupixent to for COPD.

  The approvals are based on results from the phase 3 BOREAS and NOTUS trials, which showed that Dupixent reduced moderate or severe exacerbations over 52 weeks of treatment by 30% and 34% respectively, compared to placebo.

  The nods cover patients already on a combination of an inhaled corticosteroid, a long-acting beta2-agonist (LABA) and a long-acting muscarinic antagonist (LAMA), or on a combination of a LABA and a LAMA if corticosteroids are not appropriate.

  “One in four people with COPD live in China, and many patients are unable to control their disease with standard of care treatments and experience repeated hospitalizations from exacerbations,” George Yancopoulos, M.D., Ph.D., Regeneron’s chief scientific officer, said in a release.

  China has approved Dupixent in three other indications. Dupixent was first approved by the FDA in 2017 to treat eczema.

UPDATED: Tuesday, September 24 at 5:50 p.m.

• Amgen’s thyroid eye disease med Tepezza scored approval in Japan, making it the first drug in the country cleared to treat the disease.

  Japan’s Ministry of Health, Labour and Welfare is the first regulator in Asia to approve Tepezza, which Amgen took on through its $27.8 billion Horizon Therapeutics buyout last year. The med is also approved in the U.S., Brazil and Saudi Arabia, and it's under review in Europe, Canada and Australia.

  Approximately 25,000 to 35,000 people in Japan suffer from the progressive autoimmune disease in both its active and chronic forms, which can cause eye bulging, double vision, eye pain and swelling. Amgen is currently running a separate trial to evaluate the efficacy of Tepezza in chronic thyroid eye disease patients in Japan. 

• Eli Lilly’s Kisunla received a Japanese approval to treat certain Alzheimer’s disease patients, marking the drug’s second worldwide approval.

  Kisunla was cleared to threat adults with early symptomatic Alzheimer’s disease, including those with mild cognitive impairment and those in the mild dementia stage with confirmed amyloid pathology. 

  The drug targets amyloid plaques that can lead to memory and thinking issues associated with the disease. In studies, Kisunla reduced amyloid plaques on average by 61% at six months and 80% by twelve months and significantly slowed clinical decline in patients who were less advanced in their disease, as well as in the overall disease population. The FDA was the first to approve the medicine in July. 

• Takeda, too, secured clearance from the health regulators in its home country for colorectal cancer therapy Fruzaqla.

  The drug is specifically approved to treat advanced or recurrent colorectal cancer that is not curable or resectable and has progressed after chemotherapy. In a phase 3 trial, patients received consistent benefits from Fruzaqla use regardless of the type of therapy they previously received.

  Fruzaqla scored FDA approval last year and won a nod from the European Commission in June. Under its licensing deal with Hutchmed, Takeda has an exclusive worldwide license to further develop, commercialize and manufacture the med outside of mainland China, Hong Kong and Macau. 

• Nxera Pharma (formally known as Sosei Group) can now market its insomnia drug Quviviq in Japan after winning approval from the country’s regulators.

  In a recent phase 3 study in Japanese patients, the sleep drug significantly improved total sleep time and time to sleep onset. Some 20% of adults in the country suffer from insomnia, which is characterized by persistent challenges in falling asleep and staying asleep and can cause impairment in daytime functioning.

  Nxera secured rights to the drug in the Asia Pacific region after buying Idorsia’s business units in Japan and Korea last year. Switzerland-based Idorsia brought Quviviq to the U.S. and European markets in 2022 

UPDATED: Monday, September 23 at 12:50 p.m.

• After winning an FDA approval last year, Ipsen’s Kayfanda (or Bylvay in the U.S.) has now scored approval from the European Commission under the exceptional circumstances regulatory route.

  The med was cleared to treat cholestatic pruritus in Alagille Syndrome (ALGS) in patients ages 6 months and older. ALGS is an inherited, rare disorder that can affect multiple organs and is estimated to have a global incidence rate of 3 in every 100,000 births, according to Ipsen. Severe pruritus (or itching) is a common symptom of the disease and can cause sleep disturbances for children and their caregivers.

  Ipsen backed its regulatory submission up with data from its ASSERT trial, which is the only phase 3 study ever conducted in the disease. In the study, the drug showed statistically significant improvements in pruritus as well as in several sleep parameters. An exceptional circumstances nod can be granted to a treatment for rare conditions in Europe. 

• Ipsen is on a European approval roll, also collecting a nod from the European Commission for Iqirvo as a treatment for primary biliary cholangitis.

  The drug was specifically approved for use in combination with a ursodeoxycholic acid (UDCA) to treat those with an inadequate response to UDCA or on its own for patients who can’t tolerate UDCA. 

  Ipsen gained worldwide rights to the drug in territories besides China, Hong Kong, Taiwan and Macau in a licensing deal with Genfit back in 2021 and secured FDA approval in June. Now, Genfit should receive 26.5 million euros ($29.5 million) in milestone payments.

  The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion on the med in July. The first-in-class oral med is a significant innovation for the rare liver disease, which primarily impacts women and can worsen over time without treatment. 

• The FDA accepted Leo Pharma’s bid for approval of its delgocitinib cream as a treatment for moderate to severe chronic hand eczema who don’t respond fully to topical corticosteroids.

  The regulatory submission was based on results from Leo’s phase 3 program that tested twice-daily use of the topical therapy against a placebo cream vehicle in two studies. Both studies met primary and secondary endpoints of itch and pain reduction and other hand eczema measures across 16 weeks. 

  Leo expects the FDA’s review to wrap up in the second half of 2025. 

• Meanwhile in Europe, Leo Pharma also announced that its delgocitinib cream is now approved in the European Union, where it’s branded as Anzupgo.

  The European Commission nod makes the cream the first topical treatment specifically indicated to treat certain adult patients with moderate to severe chronic hand eczema. Anzupgo is a topical pan-JAK inhibitor that inhibits the activation of JAK-STAT signaling, which plays a major role in the pathogenesis of chronic hand eczema. 

  Leo gained exclusive development and commercialization rights to the drug across dermatological indications worldwide, except for in Japan, from Japan Tobacco in 2014. 

UPDATED: Friday, September 20 at 1:30 p.m.

• As Astellas awaits an FDA decision on its gastric cancer med, Vyloy, following an earlier rejection, the European Commission has approved the med.

  European officials cleared the drug for use in combination with certain chemotherapies as a first-line treatment for patients with locally advanced unresectable or metastatic HER2-negative gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors are claudin (CLDN) 18.2 positive.

  Vyloy is the first med that's approved to specifically treat gastric tumor cells that express the CLDN18.2 biomarker, a hot target in cancer treatment. The European Commission’s blessing follows a world-first approval in Japan and a recent thumbs up in the U.K., plus an endorsement from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) in July.  

  In January, the U.S. FDA turned away Astellas' application based on unresolved deficiencies at a third-party manufacturing facility. However, the FDA had no concerns about the drug’s clinical data and has since accepted the company’s resubmitted application, assigning the prospect a target action date of November 9.

  Astellas picked up the med with its $1.4 billion purchase of Ganymed Pharmaceuticals in 2016. In phase 3 studies, Vyloy helped patients achieve statistically significant improvements in progression-free survival (PFS) and overall survival when stacked up against standard of care chemotherapies.

  More specifically, in the SPOTLIGHT trial, the chemo-Vyloy regimen was associated with a median progression-free survival duration of 10.61 months compared to 8.67 months for the control group. The GLOW trial, which used a different chemotherapy regimen along with Vyloy, yielded a median PFS of 8.21 months for the treatment group versus 6.8 months for the control group.

UPDATED: Thursday, September 19 at 10:10 a.m.

• Daiichi Sankyo's Vanflyta has won reimbursement backing (PDF) from the U.K.'s National Institute for Health and Care Excellence (NICE) for treating newly diagnosed FLT3-ITD-positive acute myeloid leukemia (AML) in England and Wales. The coverage decision follows a March approval by the Medicines and Healthcare products Regulatory Agency in the indication. 

  Vanflyta is a competitor to Novartis' Rydapt, which is approved for a broader population of FLT3-mutated AML. FLT3-ITD mutations account for about 80% of FLT3 abnormalities, or about a quarter of all AML cases. 

  NICE considered findings from the QuANTUM-First trial, which showed Vanflyta and chemotherapy reduced the risk of death by 22% versus chemo alone. Without a head-to-head trial, NICE used indirect comparisons to suggest that “there is no difference in how long people having [Vanflyta] live, or how likely it is that their AML will come back, compared with [Rydapt].”

• Xspray Pharma plans to find out if the third time is the charm for its Dasynoc, a tweaked version of Bristol Myers Squibb's leukemia drug Sprycel. Following an FDA rejection in 2023 and then in July 2024, Xspray said it plans to refile Dasynoc in the fourth quarter this year. 

  In the July complete response letter, the FDA requested that Xspray provide additional information to doctors and users to avoid confusion about the proper dosing of Dasynoc. The agency also noted that the inspection of a third-party manufacturing facility was not complete at the time. 

  Before a resubmission, the agency is asking that Xspray adjust Dasynoc's tablet strengths to reduce the risk of medication errors, and it requested new batches be produced, according to the company. Xspray has started making those test batches and will “provide further clarification on the manufacturing process” to align with the FDA's requirements, the company said.

  Compared with Sprycel, Dasynoc can be given at a lower dose because of better solubility, according to Xspray.

UPDATED: Wednesday, September 18 at 9:40 a.m.

• Five years after an adult approval to treat chronic rhinosinusitis with nasal polyposis, Sanofi and Regeneron's Dupixent has won an FDA approval to treat adolescents ages 12 to 17 years old with the same disease. The IL-4/13 antibody is the first biologic specifically approved for this patient group.

  Though systemic steroids and surgery are the standard treatment for these adolescents, many patients may still not have their symptoms—such as nasal congestion and reduced sense of smell and taste—under control. By Sanofi and Regeneron's estimate, about 9,000 adolescents in the U.S. live with inadequately controlled chronic rhinosinusitis with nasal polyps. 

  The approval is supported by efficacy data from the adult populations in the phase 3 SINUS-24 and SINUS-52 trials. In the studies, Dupixent significantly improved nasal congestion/obstruction severity, nasal polyp size and sense of smell, while also reducing the need for systemic corticosteroids or surgery at 24 weeks, compared to placebo. Additional pharmacokinetic data from adults in this indication, and from adult and adolescent patients with asthma, plus safety data in adolescents with asthma, were also considered.

• With a new FDA approval, Merck & Co.'s Keytruda, used in tandem with chemotherapy, will go up against Bristol Myers Squibb's Opdivo-Yervoy combo in the rare cancer of malignant pleural mesothelioma.

  Malignant pleural mesothelioma is a type of cancer that starts mostly in the linings of the chest and lungs. Of an estimated 30,000 new malignant mesothelioma cases worldwide in 2022, about 75% are pleural mesothelioma.

  Keytruda made its case with positive results from the phase 2/3 In KEYNOTE-483 trial. At the final analysis of the study, Keytruda and chemo reduced the risk of death by 21% versus chemo alone. 

  Patients in the Keytruda arm lived a median 17.3 months, versus 16.1 months for control. The PD-1 inhibitor also prolonged the time to tumor progression or death and increased the tumor response rate. 

UPDATED: Thursday, September 12 at 1:45 p.m.

• After passing an expected FDA action date of Sept. 7, Avadel Pharmaceuticals has confirmed that its bid to expand the approval for its narcolepsy drug is still under review by the U.S. regulator.

  Avadel had been hoping to secure a green light for Lumryz—an extended-release rival to Jazz Pharma’s Xyrem—for the treatment of cataplexy (sudden loss of muscle tone) or excessive daytime sleepiness in kids with narcolepsy ages 7 years and older.

  In a filing, Avadel said it would work with the FDA “for the remainder of the review process.” The company did not elaborate on when the FDA is expected to make an approval decision.

  Avadel’s drug first won a tentative FDA approval to fight cataplexy or excessive daytime sleepiness in adult narcolepsy patients back in 2022. Lumryz snagged a full endorsement from the U.S. drug regulator the following year, following Avadel’s victory in a patent lawsuit against Jazz last February.

  Still, Jazz didn’t take the defeat lying down. In June of 2023, the Dublin, Ireland-based company mounted a new lawsuit against the FDA, challenging the regulator for its approval of Lumryz. 

UPDATED: Friday, September 6 at 11:00 a.m.

• After receiving a scare earlier this week, Advanz Pharma’s rare liver disease drug Ocaliva will remain on the European market—for now.

  On Thursday, Advanz said that the General Court of the European Union temporarily suspended an earlier decision from the European Commission (EC) to revoke Ocaliva’s conditional marketing authorization for second-line treatment of Primary Biliary Cholangitis (PBC). PBC is an autoimmune disease in which the bile ducts become inflamed and are slowly destroyed.

  The temporary suspension of the EC’s verdict means Ocaliva will continue to be available for new and existing PBC patients across EU member states and in Iceland, Lichtenstein and Norway, Advanz said in a release.

  Just two days before the General Court’s stay, Advanz revealed that the EC had revoked the drug’s conditional marketing nod. The EC’s decision hinged on a recommendation from Europe’s Committee for Medicinal Products for Human Use (CHMP) following a reassessment of Ocaliva’s benefit-risk profile in PBC. The decision wasn’t based on any safety concerns, Advanz said.

  At the time, Advanz said it disagreed with the EC’s decision and would weigh “all options” to protect access to Ocaliva, which was conditionally approved by the European Medicines Agency in December 2016.

  Advanz argued that in making its recommendation, CHMP failed to consider more than seven years of real-world evidence on the drug. Instead, the agency relied on one analysis from a single trial that had “multiple limitations,” Advanz contended.

  Advanz snagged the rights to Ocaliva outside the U.S. for $405 million in 2022. The U.K.-based company picked up the rights from Intercept Pharmaceuticals after a 2020 FDA rejection for Ocaliva in MASH patients with liver fibrosis. As part of the deal, Intercept said at the time that a “majority” of its employees outside the U.S. would transfer over to the Advanz team. 

UPDATED: Tuesday, September 3 at 10:50 a.m.

• Joining Pfizer and Moderna, Novavax has gained regulatory authorization for its updated COVID-19 vaccine in the U.S.

  The new Novavax vaccine targets JN.1, which is the "parent strain" of currently circulating variants in the U.S., Novavax CEO John Jacobs said in the company's release.

  Novavax plans to make pre-filled syringes of its vaccine available at "thousands" of locations in the U.S., the company said.

  The FDA's emergency use authorization of the vaccine closely followed the agency's endorsement of updated COVID-19 shots from Pfizer and Moderna.

• Across the pond, the European Commission has approved a new use for the combination of Merck's Keytruda with Pfizer and Astellas' Padcev. 

  In Europe, the combo is now approved to treat first-line unresectable or metastatic urothelial carcinoma in adults. 

  The approval is based on results from the KEYNOTE-A39 trial, which showed that the combo produced statistically significant and clinically meaningful results in overall survival and progression-free survival compared with platinum-based chemotherapy. At a median follow-up of 17.3 months, the combo slashed patients' death risk by 53% and reduced PFS risk by 55%.

  “For the first time in decades, adult patients with unresectable or metastatic urothelial carcinoma have the option of a potential new first-line standard of care that may help extend their lives,” Merck Research Laboratories' SVP and head of oncology global clinical development, Dr. Marjorie Green, said in a statement.

UPDATED: Thursday, August 29 at 9:00 a.m.

• GSK's respiratory syncytial virus (RSV) vaccine Arexvy has secured an approval from the European Commission to be used in adults ages 50 to 59 who have a medical condition that increases their risk from RSV disease. 

  Such medical conditions include asthma, heart failure and diabetes, and the new label expansion covers some 20 million Europeans, according to a press release from GSK. Previously, the vaccine was approved for adults 60 and older for the prevention of lower respiratory tract disease (LRTD) caused by RSV.

  "RSV infection can have a significant impact on the health of older adults and particularly those with certain existing medical conditions, which can add pressure onto healthcare systems," GSK chief scientific officer Tony Wood said in a statement. "As we enter the RSV season, we are pleased to be the first to deliver a vaccine to help protect more people in Europe from RSV-LRTD.”

  Regulators in the U.S. granted a similar label expansion for the vaccine in June.

UPDATED: Wednesday, August 28 at 9:55 a.m.

• Astellas has gained a nod in Europe for Padcev (enfortumab vedotin) in combination with Merck’s Keytruda (pembrolizumab) for the first-line treatment of urothelial cancer. The combo is the first treatment in the indication in 40 years to display superiority to standard-of-care chemotherapy.

  A phase 3 trial showed Padcev plus Keytruda kept patients alive for an average of 31.5 months compared to 16.1 months for platinum-containing chemo, equating to a 53% reduction in the risk of death. Padcev was co-developed by Seagen, which was bought out by Pfizer last year.

  The FDA signed off on the combo treatment in the indication in April of last year. The Padcev/Keytruda pairing also was approved by the FDA in December of 2023 to treat advanced bladder cancer. 

• GSK’s Nucala (mepolizumab) has secured approval in Japan to treat adults with chronic rhinosinusitis with nasal polyps (CRSwNP). The anti-inflammatory treatment becomes the only biologic in the country with a four-week dosing schedule for the disorder.

  It is the third indication in Japan for the monoclonal antibody, which targets interleukin-5 (IL-5). Nucala is also approved in Japan for some forms of asthma and for eosinophilic granulomatosis with polyangiitis (EGPA). The FDA endorsed Nucala for asthma in 2015, for EGPA in 2017 and for CRSwNP in 2021.

  People with CRSwNP experience nasal obstruction, loss of smell, facial pressure, sleep disturbance and nasal discharge. In Japan, an estimated 2 million people suffer from chronic rhinosinusitis, of which about 200,000 are subject to surgery due to nasal polyps, GSK said.

  Nucala sales reached (PDF) 1.66 billion pounds ($2.1 billion) in 2023.

UPDATED: Tuesday, August 27 at 10:51 a.m.

• Roche’s C5-blocking antibody PiaSky, also known as crovalimab, has passed muster with European regulators to treat adults and kids ages 12 and older with the rare blood condition paroxysmal nocturnal hemoglobinuria (PNH).

  To qualify for the med, PNH patients must weigh at least 40 kg (88 pounds), and they must either be new to or have previously been treated with C5 inhibitors, Roche said Tuesday.

  In patients with PNH—which can be life-threatening—red blood cells are destroyed by the complement component of the immune system. The condition causes symptoms like anemia, fatigue and blood clots. PNH can also lead to kidney disease down the line, Roche explained.

  Many PNH patients continue to rely on frequent intravenous infusions to treat their disease, whereas Roche’s med has now become the first monthly subcutaneous therapy approved for PNH in the European Union (EU).

  The European Commission based its approval on Roche’s Commodore 2 study, which looked at patients with PNH who hadn’t previously tried a C5 inhibitor. The trial found that once-a-month PiaSky helped patients achieve disease control while remaining well-tolerated.

  In the trial, Roche’s drug was also non-inferior to eculizumab, which is considered a current standard of care C5 inhibitor for PNH. 

  Besides Europe, the drug has been approved in the U.S., China and Japan.

• Despite Legend Biotech’s Carvykti being developed in China, the CAR-T therapy has only just now won approval in its home country.

  On Tuesday, China’s National Medical Products Administration (NMPA) signed off on the cell therapy to treat adults with relapsed or refractory multiple myeloma whose disease progressed after receiving at least three prior lines of therapy, Legend said in a securities filing.

  Those prior lines of treatment can include a proteasome inhibitor and immunomodulatory agent, the company added.

  The approval of Carvykti in China was based on data from Legend’s ongoing phase 2 study Cartifan-1, which is being run in-country.

  Carvykti, which Legend partners on with Johnson & Johnson, was first approved for multiple myeloma in the U.S. back in February of 2022. 

• Despite a regulatory setback in 2023, ARS Pharmaceuticals is making good on its promise to deploy a needle-free alternative to ubiquitous epinephrine autoinjectors.

  Following the U.S. approval of the company’s epinephrine nasal spray Neffy earlier this month, ARS has picked up another nod for its anaphylaxis med for kids and adults in Europe.

  Across the pond, the drug will be known as EURneffy, ARS explained in a release.

  With the green light, EURNeffy now becomes the first needle-free treatment option for kids and adults in the EU with severe allergies, many of whom “may not carry, or delay use of an injectable adrenaline product,” Antonella Muraro, M.D., Ph.D., professor of food allergy at the University of Padua, said in a statement.

  To hear ARS’ co-founder and CEO Richard Lowenthal tell it, EURneffy’s compact size, needle-free design and longer, less temperature-sensitive shelf-life could boost the odds that patients will both carry and administer adrenaline in the case of severe allergic reactions.

  ARS figures EURneffy will become available in certain EU member states starting in 2024’s fourth quarter. Marketing in the bloc will be handled by a “pharmaceutical company with an already established commercial footprint in Europe,” ARS said.

  Neffy won approval in the U.S. in early August. The drug is currently poised for an October launch in the States. 

UPDATED: Monday, August 26 at 11:25 a.m.

• Merck & Co.'s Winrevair has won European Commission (EC) approval as part of combinations with other therapies to treat pulmonary arterial hypertension (PAH) in adult patients with World Health Organization (WHO) Functional Class 2 to 3 disease. The EC nod follows an FDA approval for the drug in PAH in March.

  The drug's approved indications are slightly different in the U.S. and Europe. In the U.S., Winrevair is cleared to increase exercise capacity, improve WHO functional class and reduce the risk of clinical worsening events. In EU, the indication is simply to increase exercise capacity. 

  As the first therapy targeting the activin signaling pathway, Winrevair won is EC approval based on data from the phase 3 STELLAR trial. In patients with WHO Group 1 functional class 2 or 3 disease, Winrevair led to a statistically significant and clinically meaningful improvement in patients' six-minute walk distance compared with placebo.

  Merck got Winrevair from its $11.5 billion acquisition of Acceleron Pharma in 2021. Analysts have come up with a wide range of peak sales estimates, ranging from around $2 billion to $11 billion.

• Rhythm Pharmaceuticals' application to expand its rare genetically-caused obesity med Imcivree to younger patients as young as 2 years old has been put under FDA priority review, with a decision set for Dec. 26. 

  The drug is currently approved in the U.S. for adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity due to POMC, PCSK1 or LEPR deficiency. 

  In a phase 3 study conducted in 12 patients ages 2 to 6 years, treatment with Imcivree achieved the study's primary endpoint by enabling a 3.04 mean reduction in patients' BMI-Z scores and an average 18.4% reduction in BMI. The BMI-Z score a measure of body mass index deviations from what is considered normal.

  The European Commission in July expanded the drug's age range to include children as young as 2 years old.

• The European Commission also approved Celltrion's SteQeyma, a biosimilar version of Johnson & Johnson's Stelara, across multiple chronic inflammatory diseases in gastroenterology, dermatology and rheumatology.

  SteQeyma is Celltrion's seventh biosimilar approved in Europe.

UPDATED: Thursday, August 22 at 4:55 p.m.

• Bristol Myers Squibb’s Opdivo and Yervoy combo is up for a potential label expansion into first-line treatment of unresectable hepatocellular carcinoma (HCC).

  The FDA accepted the company’s application for the new indication based on phase 3 trial results that demonstrated the combo's statistically significant and clinically meaningful improvement in overall survival compared to investigator’s choice of Eisai and Merck’s Lenvima or Bayer’s Nexavar.

  The Opdivo-Yervoy combo is already approved as a second-line treatment for advanced HCC, which is the most common type of primary liver cancer.

  The FDA is set to decide on the expansion bid by April 21.

• Just after collecting U.K. approval to treat ulcerative colitis, AbbVie’s Skyrizi also bagged a thumbs up from the National Institute for Health and Care Excellence (NICE).

  The nod makes the drug available to an estimated 296,000 ulcerative colitis patients in the U.K and comes one day after the Medicines and Healthcare Products Regulatory Agency (MHRA) cleared Skyrizi’s use in certain adult UC patients.

  With the approval, Skyrizi can now treat both forms of inflammatory bowel disease in England. NICE recommended the drug's use in Crohn’s disease last year. 

UPDATED: Tuesday, August 20 at 9:00 a.m.

• AbbVie and Genmab's Tepkinly has gained its second approved indication in Europe. 

  The European Commission (EC) conditionally approved the subcutaneous bispecific antibody to treat adults with relapsed or refractory (R/R) follicular lymphoma (FL) and after two or more lines of prior systemic therapy. There are around 13,000 cases of FL each year in Western Europe, according to an estimate cited by AbbVie.

  The approval was supported by results from the Phase 1/2 EPCORE NHL-1 clinical trial. The open-label, single-arm trial evaluated the the drug as a monotherapy in adults with R/R FL after two prior lines of systemic therapy. Investigators recorded an overall response rate of 83% and a complete response rate of 63% among patients enrolled in the study.

  The FL nod follows a September 2023 EC thumbs up for the medicine to treat adults with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy.

• In China, AstraZeneca's Fasenra has gained an approval from the National Medical Products Association (NMPA) as a maintenance treatment for people 12 and older with severe asthma. 

  The approval was supported by results from the Phase 3 MIRACLE study, which enrolled patients in China, South Korea and the Philippines. In the study, the AstraZeneca drug delivered a "statistically significant and clinically meaningful" 74% reduction in patients' annualized asthma exacerbation rate when added to standard care, the company said in a Tuesday press release.

  About 3 million people suffer from severe asthma in China, according to an estimate cited by the drugmaker.

UPDATED: Monday, August 19 at 8:05 a.m.

• AstraZeneca and Daiichi Sankyo's Enhertu has gained a "Breakthrough Therapy" designation from the FDA as part of the companies' effort to expand the drug's reach.

  The FDA granted the designation to Enhertu as a potential treatment for certain patients with HER2-low or HER2-ultralow breast cancer. The designation is intended to speed the development and review of promising medicines that address diseases with significant unmet need.

  Back in June, the partners shared the results of the DESTINY-Breast06 at the American Society of Clinical Oncology annual meeting in Chicago.

  While a prior trial, DESTINY-Breast04, enabled the partners to score a landmark approval in the "HER2-low" patient class, the results shared at ASCO fueled conversation about reaching patients with even lower levels of HER2 expression.

  This is Enhertu's eighth "Breakthrough Therapy" designation from the FDA.

• As Bristol Myers Squibb works to grow its CAR-T drug Breyanzi, the company's regulatory efforts in Europe are bearing fruit.

  Monday, Bristol Myers said the European Medicines Agency (EMA) validated its application for Breyanzi as a treatment for adults with relapsed or refractory follicular lymphoma (FL) who have received at least two lines of systemic therapy.

  The company's EMA filing leverages results from the TRANSCEND FL study, which supported the drug's FDA approval in FL three months ago.

UPDATED: Thursday, August 15 at 3:20 p.m.

• Theramex’s Yselty will also soon be available in the U.K. following a recommendation from England’s National Institute for Health and Care Excellence (NICE) as a treatment for symptoms of moderate to severe uterine fibroids.
  
  Yselty is a once-a-day GnRH antagonist tablet that reduces bleeding linked to uterine fibroids and shrinks the fibroids, which are non-cancerous growths in or around the uterus. Around 66% of women develop at least one uterine fibroid over their lifetime and 33% of women get symptoms from the fibroids, according to NICE. That means about 30,000 adults could benefit from the coverage, the agency said. Typical treatments include hormonal treatments, which Yselty can be paired with.
  
  The drug is not approved in the U.S after its regional licensee ObsEva terminated its license agreement following an FDA rejection in 2022, leaving the company to restructure and cut 70% of its workforce. 

• The FDA will undertake a speedy review of AstraZeneca’s Imfinzi as a limited-stage small cell lung cancer (LS-SCLC) treatment in certain patients after accepting the company’s application under a priority review track and assigning a decision date over the fourth quarter of this year.

  AZ is going for a label expansion to include LS-SCLC patients whose disease has not progressed following platinum-based concurrent chemotherapy. In a phase 3 study, Imfinzi reduced the risk of death by 27% compared with placebo and cut the risk of disease progression or death by 24% versus placebo.

  SCLC is a highly aggressive cancer that often progresses rapidly and carries a five-year survival rate of 15% to 30% after diagnosis, according to AZ.  

• NICE also deemed Myovant’s oral treatment Orgovyx a cost-effective option for patients with hormone-sensitive prostate cancer (HSPC) in a final draft guidance.

  The treatment offers an alternative to the standard injectable androgen deprivation therapy to address some of the 44,000 new cases of prostate cancer diagnosed across the U.K. each year. The cancer is the second most common type in men in the region, according to the charity Prostate Cancer UK.

  NICE’s nod follows a June 2022 marketing authorization from the Medicines and Healthcare products Regulatory Agency (MHRA). The NICE backing cover Orgovyx's use either alongside or before radiotherapy for high-risk localized or locally advanced HSPC. 

UPDATED: Wednesday, August 14 at 10:00 a.m.

• The European Commission has approved AstraZeneca's Imfinzi-Lynparza-chemo combo as first-line treatment for endometrial cancer that's mismatch repair proficient (pMMR). And the Imfinzi-chemo regimen without Lynparza was cleared to treat patients with mismatch repair deficient (dMMR) disease.

  The approvals matched findings from the DUO-E trial. In the trial, the Lynparza-containing regimen reduced the risk of disease progression or death for patients with pMMR disease by 43% versus the control arm, whereas the sans-Lynparza regimen only reduced that risk by 9%. By comparison, Imfinzi and chemo could already pare down the risk of progression or death by 58% in dMMR patients, and the extra addition of Lynparza posted a nearly identical benefit at 59%.

  Before EU's approval, the FDA had in June only approved Imfinzi plus chemotherapy in dMMR patients. In a statement to Fierce Pharma at that time, an AZ spokesperson said the company will continue to follow overall survival data and discuss next steps with the FDA.

• U.K.'s drug regulators have approved Astellas' first-in-class Vyloy, used alongside chemotherapy, to treat gastric or gastroesophageal junction cancer that's CLDN18.2-positive and HER2-negative. 

  In approving the CLDN18.2-targeted drug, the Medicines and Healthcare products Regulatory Agency noted improved overall survival for Vyloy's combinations with two different chemo regimens versus chemo alone in two phase 3 trials, SPOTLIGHT and GLOW. 

  Before the U.K. nod, Japanese authorities have approved Vyloy in March, and drug reviewers at the European Medicines Agency also recommended an approval in late July, teeing up an official green light by the European Commission. 

  The FDA rejected Vyloy at the beginning of 2024, citing manufacturing issues. Astellas has refiled, and the FDA has set a new target decision date on Sept. 9. 

UPDATED: Tuesday, August 13 at 12:07 p.m.

• After snagging two approvals in China last year, AstraZeneca and Daiichi Sankyo's antibody-drug conjugate (ADC) Enhertu has picked up a third nod in the country.

  China's National Medical Products Administration (NMPA) this week granted a conditional approval to Enhertu as a monotherapy to treat adults with locally advanced or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma who've tried two or more prior treatments, the partners said Tuesday. 

  NMPA based its decision on positive results from the midstage DESTINY-Gastric06 trial, though full approval will depend on whether a confirmatory study can demonstrate clinical benefit, AZ and Daiichi said. 

  According to the partners, more than a third of global gastric cancer cases crop up in China, with roughly 65% of patients presenting with advanced disease at the time of diagnosis. 

  The green light marks the third approval for the partners' ADC in China, following rapidfire approvals last year in HER2-positive metastatic breast cancer and HER2-low metastatic breast cancer.

  Elsewhere, Enhertu is approved for advanced or metastatic gastric cancer in more than 45 countries, including the United States, Japan and across the European Union. 

• The FDA has granted breakthrough therapy designation to Amgen and AstraZeneca’s Tezspire as an add-on maintenance treatment for patients with moderate to very severe chronic obstructive pulmonary disease (COPD). Amgen announced the milestone in its quarterly earnings report.

  The companies recently provided results from a phase 2 study that showed that Tezspire reduced the rate of moderate or severe COPD exacerbations by 17% compared to placebo. Planning for a phase 3 trial is underway.

  After more than a decade without any significant treatment upgrades in the indication, Verona Pharmaceuticals gained an FDA approval in June for Ohtuvayre as an add-on and monotherapy for COPD. Next month, the FDA is expected to sign off on Sanofi and Regeneron’s Dupixent as an add-on treatment for COPD.

UPDATED: Monday, August 12 at 8:45 a.m.

• Sandoz's biosimilar version of Regeneron's Eylea has picked up FDA approval. 

  The agency approved Sandoz's Enzeevu in a 2 mg vial kit and a pre-filled syringe for intravitreal injection to improve and maintain visual acuity in patients with neovascular age-related macular degeneration (nAMD). The FDA "provisionally determined" the biosimilar qualifies for interchangeable status with the reference drug, Sandoz said in a Monday press release.

  Sandoz said "launch timing will be dependent on several factors, including the progress and outcome of pending or potential future related litigations or any potential settlements."

  Earlier this year, Sandoz bought Coherus' Lucentis biosimilar business, including ophthalmology sales and reimbursement teams. Sandoz plans to use those teams to support the Enzeevu launch.

  Besides nAMD, Regeneron's originator drug is approved for several other ophthalmology indications such as diabetic macular edema, diabetic retinopathy and macular edema following retinal vein occlusion.

• England's National Institute for Health and Care Excellence (NICE) laid out its rationale behind a trio of recent multiple myeloma decisions.

  The agency has signed off on Pfizer's Elrexfio and Johnson & Johnson's Tecvayli for certain patients with multiple myeloma. Specifically, the drugs are endorsed for patients who've tried at least 3 prior therapies, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody.

  Since the companies compared their drugs against the combination of pomalidomide plus dexamethasone (PomDex) in trials, NICE recommended them only for patients "would be eligible for pomalidomide plus dexamethasone at this stage in the pathway." Patients who've already been treated with PomDex would not be eligible under NICE's guidance.

  In addition, NICE said it was unable to recommend Sanofi's Sarclisa in combination with PomDex for patients with relapsed or refractory multiple myeloma who have already been treated with lenalidomide and a proteasome inhibitor.

  The agency received two appeals of that decision, which will be heard on September 23, according to a Friday press release.

UPDATED: Friday, August 9 at 12:15 p.m.

• Those with severe allergies in the U.S. have a new option for emergency anaphylaxis treatment in ARS Pharmaceuticals’ neffy nasal spray.
  
  Neffy is the first FDA approved epinephrine product that is not administered by injection, representing a new option to EpiPens. Epinephrine is the only life-saving anaphylaxis treatment, according to the FDA.
  
  Four studies in adults and one in children showed that neffy delivered comparable epinephrine blood concentrations to EpiPen and similar blood pressure and heartrate increases, two “critical effects” of epinephrine treatment. The drug is a single-dose spray that’s administered into one nostril, but it can be dosed a second time if symptoms worsen or don’t improve.

  The approval came ahead of schedule and followed an FDA Complete Response Letter that required ARS to conduct additional research in support of its filing.

UPDATED: Wednesday, August 7 at 8:30 a.m.

• In quick succession, Exelixis has provided another update on its cancer drug Cabometyx. This time, the company said the phase 3 CONTACT-02 trial did not meet one of its primary endpoints.

  The study is testing the combination of Cabometyx and Roche's Tecentriq against a second novel hormonal therapy (NHT) in patients with metastatic castration-resistant prostate cancer and measurable soft-tissue disease who progressed after a prior NHT. The trial previously met one primary endpoint of progression-free survival in a predefined patient population. But Tuesday, Exelixis said the trial fell short on the overall survival (OS) endpoint. 

  The OS endpoint "continued to favor the combination" but did not reach statistical significance at the final analysis, Exelixis said in an update. Nonetheless, the company is planning an FDA submission in pursuit of an approval in the use this year.

• Across the pond, England's National Institute for Health and Care Excellence (NICE) recommended Theramex's Eladynos as an option for the treatment of osteoporosis for cases with a very high risk of fracture.

  Under the agency's guidance, more than 14,000 people stand to benefit from the new treatment option, NICE said in a release.

  The drug will be available on NHS England within three months, NICE said.

• In its second-quarter update, BeiGene said that the FDA "has deferred approval for tislelizumab in first-line unresectable, recurrent, locally advanced, or metastatic" esophageal squamous cell carcinoma (ESCC). The drug is approved as Tevimbra in certain second-line ESCC cases. 

  BeiGene said "a delay in scheduling clinical site inspections" resulted in the FDA's deferral.

  The company's first-line application previously carried an FDA action date in July, so a regulatory hiccup was expected given that the date had passed.

UPDATED: Tuesday, August 6 at 10:50 a.m.

• The FDA has accepted Exelixis’ application for Cabometyx (cabozantinib) for the treatment of adults with previously treated pancreatic neuroendocrine tumors (pNET). The application covers patients with locally advanced/unresectable or metastatic, well- or moderately differentiated pNET or differentiated extra-pancreatic NET (epNET).

  The FDA also granted orphan drug designation to cabozantinib and established a target decision date of April 3, 2025.

  Cabometyx was originally approved in 2016 for advanced renal cell carcinoma. That green light was followed by FDA nods to treat hepatocellular carcinoma in 2019 and thyroid cancer in 2021. The California company reported that sales of Cabometyx reached $1.6 billion last year.

  Neuroendocrine tumors begin in the specialized cells of the body’s neuroendocrine system. In the U.S., it is estimated that 161,000 to 192,000 people are living with unresectable, locally advanced or metastatic NET. 

• The European Commission has granted marketing authorization to UCB’s two new presentations of Bimzelx (bimekizumab)—a pre-filled syringe and a pre-filled pen—both at a dose of 320 mg. The new options provide convenience for patients prescribed for a 320 mg dose. Previously, Bimzelx was only available in a 160 mg injection.   

  In Europe, Bimzelx is indicated for people with moderate to severe plaque psoriasis, active psoriatic arthritis with coexistent moderate to severe plaque psoriasis, and active moderate to severe hidradenitis suppurativa. It was approved in the U.S. for plaque psoriasis in October of last year.

UPDATED: Friday, August 2 at 8:05 a.m.

• After receiving a Complete Response Letter (CRL) from the FDA in April, Supernus has refiled its apomorphine infusion device (SPN-830) with the regulator. 

  The company seeks an approval for the device to provide continuous treatment of motor fluctuations (OFF episodes) in patients with Parkinson’s disease (PD). 

  The agency flagged two shortfalls in Supernus' prior filing. In a Friday release, the company said it "believes it has addressed" the FDA's questions.
  
  Besides the April 2024 rejection, the drug previously hit a wall at the FDA in 2022, when the agency sent the company a refuse-to-file notice in response to Supernus' first SPN-830 application.

• Also in the U.S., Grifols has received a label expansion for its subcutaneous immunoglobulin (SCIg) treatment Xembify. 

  With a new FDA approval, the drug can now be used by treatment-naïve patients with primary humoral immunodeficiencies (PI). The company notes that Xembify is now the first in treatment in its class with available for this patient group.

  The approval will allow PI patients to start on SCIg therapy right away, rather than first starting with an intravenous infusion, according to Grifols.

  “The Xembify label expansion eliminates the need for patients to have initial intravenous treatment, which differentiates Xembify from other SCIg therapies, plus offers patients greater convenience and flexibility with biweekly dosing,” Grifols' chief scientific innovation officer, Joerg Schuettrumpf, said in a statement. 

  The company plans to launch the drug in its new use during the next few months.

• Octapharma's Fibryga scored an additional FDA approval for fibrinogen replacement in bleeding patients with acquired fibrinogen deficiency (AFD), the company announced on Thursday.

  Fibryga is the first on-demand, virus-inactivated, human plasma-derived fibrinogen concentrate option available in the indication, according to the company. It offers a "rapid and more precise option for severe bleeding scenarios" than cryoprecipitate, the current standard of care, Octapharma added.

  Fibryga picked up its first FDA approval in 2017 to address acute bleeding episodes in adults and adolescents with congenital fibrinogen deficiency (CFD). Then in 2020, the FDA approved the product for patients under 12 in that indication.

UPDATED: Tuesday, July 30 at 10:15 a.m. 

• The FDA has granted Novartis' Scemblix a priority review as it weighs a potential label expansion for the medicine. Specifically, Novartis seeks an approval for Scemblix to treat newly diagnosed adult patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP).

  The designation means that the FDA aims to make a decision on approval within 6 months, compared with 10 months for standard reviews.

  Novartis' Scemblix is already approved to treat patients with Philadelphia chromosome-positive chronic myeloid leukemia who've tried two or more tyrosine kinase inhibitors, plus Ph+ CML-CP patients with the T315I mutation.

  The company's label expansion filing is supported by data from the ASC4FIRST trial, which showed "significantly improved molecular response and a favorable safety and tolerability profile compared to standard of care therapies," Novartis said in a release.

• Across the pond, the European Commission has signed off on Roche's filing for Vabysmo for treatment of retinal vein occlusion (RVO).

  With the approval, Roche's stalwart eye disease medicine has moved into 3 eye disease indications in Europe. Besides RVO, Vabysmo is approved to treat wet age-related macular degeneration (nAMD) and diabetic macular edema (DME) in Europe.

  "The efficacy and safety of Vabysmo has been well established in global clinical trials and is reinforced by a growing breadth of real-world evidence, with hundreds of thousands of people treated worldwide," Roche's chief medical officer and head of global product development, Levi Garraway, M.D., Ph.D., said in a statement.

  The European Commission's decision follows a label expansion recommendation from the European Medicines Agency's Committee for Medicinal Products for Human Use last month.

UPDATED: Thursday, July 25 at 8:35 a.m. 

• BioMarin's Brineura has gained FDA approval to slow the loss of ambulation in all children with neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as tripeptidyl peptidase 1 (TPP1) deficiency.
  
  Previously, the drug was approved in patients 3 and older with late infantile CLN2 disease.
  
  As the company noted in its press release, the new indication covers all children with CLN2 disease—regardless of whether they are symptomatic or presymptomatic.
  
  "Today's approval represents a significant step forward in enabling children to be treated with BRINEURA as early as possible, when we can have the greatest impact in altering the natural course of disease," BioMarin's worldwide R&D head, Hank Fuchs, M.D., said in a statement.
  
  The disease is "characterized by a rapid onset of neurodegenerative symptoms," Fuchs explained.
  
  In an open-label Phase 2 study called Study 190-203, CLN2 patients treated with the drug had a slower decline in motor function and disease onset than would be expected under the natural course of the disease. The study included 8 patients who were younger than the age of 3.
   
• In England, Boehringer Ingelheim's stroke drug Metalyse has gained a positive recommendation from the National Institute for Health and Care Excellence (NICE).
  
  NICE acknowledged that the drug works as well as alteplase, a NICE-recommended treatment option also marketed by Boehringer in the United Kingdom, in breaking up and preventing blood clots in the wake of a stroke.
  
  But since Metalyse is cheaper than alteplase, its use could save the National Health Service millions of pounds, NICE said in a release this week.
  
  Metalyse is given early during a stroke and prompts patients to produce plasmin, which breaks down blood clots, according to the agency.
   

UPDATED: Tuesday, July 23 at 5:35 p.m. 

• The U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) granted another approval to Novo Nordisk’s Wegovy, expanding the med’s reach with a new indication to reduce the risk of serious cardiovascular events in obese patients.

  The indication makes Wegovy the first weight loss drug approved to prevent cardiovascular events such as stroke, heart attack and cardiovascular death in patients with a specific body mass index and an established cardiovascular disease, according to an MHRA press release.

  MHRA’s nod follows the same approval from the FDA back in March. Both approvals were based on a study of more than 17,600 participants that found Wegovy cut the risk of major adverse cardiovascular events by 20% compared with placebo.
   

• Arcutis submitted an FDA bid for another indication for its Zoryve foam, this time chasing an approval for scalp and body psoriasis in patients ages 12 and older.

  The submission marks the company’s fifth for Zoryve in less than three years, CEO Frank Watanabe said in a release. So far, the treatment is approved to target seborrheic dermatitis and to topically treat mild to moderate atopic dermatitis, plus plaque psoriasis.

  Some 40% of the estimated 9 million U.S. plaque psoriasis patients have scalp involvement, which is difficult to treat topically due to the challenge of applying drugs in areas typically covered by hair.
  
  In a trial, 67.3% of Zoryve-treated patients achieved a measure of disease improvement that looked at scalp psoriasis coverage, compared with 28.1% of those treated with a vehicle foam.
   

UPDATED: Monday, July 22 at 9:40 a.m. 

• Four years after Spravato's original FDA approval, Johnson & Johnson has filed for a label expansion for the depression nasal spray.
  
  J&J seeks FDA approval for the drug to be used as a monotherapy in adults with treatment-resistant depression (TRD). TRD occurs when patients don't respond to two or more oral antidepressants during the same depressive episode, according to J&J.
  
  Currently, the J&J drug is approved to be used in combination with an oral antidepressant.
  
  In the placebo-controlled Phase 4 TRD4005 study, the drug "showed a rapid change" in patients' depression, as measured by the Montgomery-Asberg Depression Rating Scale, J&J said in a press release.
  
  Of an estimated 280 million people living with major depressive disorder worldwide, nearly 30% have TRD, according to J&J.
  
   
• Elsewhere, J&J's multiple myeloma bispecific antibody, Tecvayli, has gained a new recommendation from England's National Institute for Health and Care Excellence (NICE).
  
  NICE endorsed the therapy to treat adults in England and Wales with relapsed and refractory multiple myeloma who've tried at least three prior treatments, who have progressed on their following therapy, and who would otherwise be given pomalidomide/dexamethasone. 
  
  The decision marks a turnaround from NICE's negative decision on Tecvayli back in early 2023.
  
   
• The FDA has approved Samsung Bioepis' Epysqli, a biosimiler version of AstraZeneca's complement inhibitor Soliris.
  
  This is Samsung Biopeis' eighth biosimilar approval at the FDA, according to a company press release.
  
  The drug is approved to reduce hemolysis in patients with paroxysmal nocturnal hemoglobinuria (PNH) and to inhibit complement-mediated thrombotic microangiopathy in those with atypical hemolytic uremic syndrome (aHUS).
  
  Samsung Bioepis did not share information Epysqli about its launch plans.
  
  The company stressed that the drug is not approved for the treatment of patients with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS).
   

UPDATED: Friday, July 19 at 11:00 a.m. 

• Eli Lilly’s weight loss powerhouse tirzepatide will take on another major market with an approval from China’s National Medical Products Administration (NMPA).

  The drug, branded in China as Mufengda, was approved for long-term weight management, according to a social media post, following a clinical trial based on Chinese diagnostic standards with a specific body mass index (BMI) cutoff point. Tirzepatide has been approved in China as a diabetes treatment since May.

  Novo Nordisk’s rival obesity med Wegovy beat Lilly to the chase in June with its NPMA nod, setting up the two GLP-1 drugmakers to duke it out in the world’s second-largest economy. A 2019 study found that 48.9% of the overall study population of 15.8 million adults in China had obesity or were overweight.
   

• GSK is looking to bring its antibody-drug conjugate Blenrep back to the European market as the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has accepted the company’s marketing application.
  
  The drug was recently withdrawn following a CHMP vote to not renew its conditional market authorization following a negative phase 3 trial for the drug as a monotherapy.
  
  This time, GSK is seeking a nod in relapsed or refectory multiple myeloma for Blenrep's combination with either Takeda’s Velcade plus steroid dexamethasone or BMS’ Pomalyst and dexamethasone following positive results from two phase 3 trials. In the studies, the therapy proved statistically significant and clinically meaningful improvements in progression-free survival compared to standard of care combos.
  
  A positive overall survival trend was observed in both studies but did not meet statistical significance by the interim analysis, the company noted in a release.
  
   
• The EMA is also reviewing Bristol Myers Squibb’s application for its Opdivo and Yervoy combo as a first-line treatment for patients with unresectable or advanced hepatocellular carcinoma (HCC) who have not received prior systemic therapy.
  
  The EMA validated the company’s application, which is based on a phase 3 study that showed significant and meaningful improvement in overall survival compared with Eisai and Merck’s Lenvima or Bayer’s Nexavar. Specifically, patients who received Opdivo and Yervoy lived 3.1 months longer than those who received either one of the two comparator tyrosine kinase inhibitors at the median, reaching 23.7 months. 
  
  BMS' Opdivo-Yervoy duo holds indications across cancer types including lung cancer, kidney cancer and colon cancer, to name a few.
  
   
• Phathom Pharmaceuticals’ Voquezna scored its third FDA approval with a nod for non-erosive gastroesophageal reflux disease (GERD)-associated heartburn.
  
  Non-erosive GERD is the largest category of the disease with an estimated patient pool of 45 million U.S. adults, 15 million of whom are treated with a prescription medicine annually. Voquezna represents a new class of acid suppression therapy and the first major innovation in the space in over three decades, according to a company press release.
  
  The pill is also approved for erosive GERD (erosive esophagitis) and to treat bacterial infection heliobacter pylori in combination with antibiotics.
   

UPDATED: Monday, July 15 at 9:30 a.m. 

• The United Kingdom's Medicines and Healthcare products Regulatory Agency (MHRA) has approved Roche's subcutaneous version of Ocrevus to treat patients with relapsing and primary progressive multiple sclerosis. 
  
  Previously approved in the U.K. as an infusion, the drug can now be given as a subcutaneous injection by a doctor or nurse every six months, MHRA said in a release.
  
  In a randomized study of 236 patients, patients who received a single dose of the injected drug had similar levels of medicine in the blood as those who received two infusions.
  
  Elsewhere, regulators in Europe granted subcutaneous Ocrevus an approval last month.
  
   
• AbbVie has submitted U.S. and European approval applications for Rinvoq in giant cell arteritis (GCA), the company said in a Monday press release.
  
  The filings are backed by data showing the drug met its endpoint of sustained remission from week 12 to week 52 in the placebo-controlled SELECT-GCA trial.
  
  GCA, an autoimmune disease of patients' medium and large arteries, can cause headaches, jaw pain and sudden vision changes.
  
   
• Health authorities in Canada have approved AstraZeneca's Tagrisso plus chemotherapy for patients with locally advanced or metastatic non-small cell lung cancer whose tumors have EGFR exon 19 deletions or exon 21 (L858R) substitution mutations.
  
  The approval is based on results from the FLAURA2 trial, which enabled recent approvals for the AstraZeneca drug in Japan and China.
  
  FLAURA 2 is a global phase 3 trial that enrolled first-line patients with locally advanced or metastatic EGFR-mutated non-small cell lung cancer. In the overall trial population, the Tagrisso and chemo pairing cut the risk of disease progression or death by 38% compared with Tagrisso alone. Tagrisso is the global first-line standard of care for these patients.
  
  "Having this new option available to Canadians living with lung cancer that effectively targets their specific type of lung cancer to help them live well longer is great news for the lung cancer community and another step in the right direction," Lung Cancer Canada executive director Shem Singh said in a statement. "We look forward to Canadians across the country being able to benefit from this treatment shortly."
   

UPDATED: Wednesday, July 10 at 9:15 a.m. 

• Arcutis' steroid-free topical PDE4 inhibitor Zoryve has picked up a label expansion to move into its third disease area.
  
  The FDA approved the drug to treat patients with atopic dermatitis down to the age of 6. Before this approval, the drug was approved for patients 9 and older with seborrheic dermatitis and for those 6 and older with plaque psoriasis.
  
  The once-daily cream can be used anywhere on the body for "any duration," the dermatology specialist said in its press release. Its benefits have been proven for up to 56 weeks, according to the company.
  
  In clinical trials, the drug demonstrated an ability to help clear patients' atopic dermatitis and to provide a "rapid and significant" improvement in itch, the most burdensome symptom of the disease, according to Arcutis.
  
   
• Across the pond, Almirall's Ebgylss has picked up a recommendation from England's National Institute for Health and Care Excellence (NICE) to treat certain patients with moderate to severe atopic dermatitis.
  
  Specifically, the drug is recommended for patients 12 and older who haven't responded to at least 1 systemic immunosuppressant, or those who can't take those drugs.
  
  The NICE recommendation follows a regulatory endorsement from the United Kingdom's Medicines and Healthcare products Regulatory Agency (MHRA) in December 2023.
  
  After a startup treatment phase, patients can benefit from a 4-week maintenance dosing schedule. 
   

UPDATED: Tuesday, July 9 at 10:40 a.m.

• AstraZeneca’s Imfinzi picked up authorization from the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) to treat certain patients with respectable non-small cell lung cancer.

  The therapy can now be used with platinum-based chemotherapy as a neoadjuvant treatment before surgery and on its own after surgery in patients with resectable NSCLC who have no known epidermal growth factor receptor mutations, ALK rearrangements or aberrations, PMLiVE reports.

  The U.K. nod is the latest in a series of recent regulatory advancements for the drug. Imfinzi was also recommended by Europe’s Committee for Medicinal Products for Human Use (CHMP) on July 1 for use in combination with AZ’s Lynparza as a treatment for certain patients with primary advanced or recurrent endometrial cancer, plus alongside chemotherapy and then on its own for patients with mismatch repair deficient disease.
   

• South Korea’s Alteogen scored an approval from the country’s Ministry of Food and Drug Safety (MFDS) for its Tergase, a recombinant hyaluronidase that can be used for “various purposes.”

  Along with its “main application” as a dermal filler removal, Tergase can also be used for orthopedics pain management or as a local anesthetic solution for eye surgery, the company said in a press release.
   

• Rare disease drugmaker Zevra Therapeutics’ Niemann-Pick disease type C candidate arimoclomal will be reviewed at a meeting of the FDA’s newly formed Genetic Metabolic Disease Advisory Committee in August ahead of the agency’s September decision date.

  The candidate holds an orphan drug designation, a fast track designation, a breakthrough therapy designation and rare pediatric disease designation from the FDA. Zevra “looks forward” to presenting its data to the committee, CEO Neil MacFarlane said in a press release.

  Zevra acquired arimoclomal from its $12.8 million Orphazyme deal after the original developer hit several setbacks with the drug, including a prior FDA rejection and failed trials in other disease areas.
   

UPDATED: Monday, July 1 at 8:51 a.m.

• Sandoz and Samsung Bioepis' Pyzchiva, a biosimilar to Johnson & Johnson's Stelara, has gained FDA approval. 
  
  The agency provisionally endorsed the biosimilar as an interchangeable product, meaning that in some cases it can be substituted at the pharmacy counter without a doctor's permission.
  
  Sandoz will commercialize the biosimilar in the U.S. under the partnership between the companies. The biosimilar is set to reach the market in February of next year thanks to a settlement the partners struck with Johnson & Johnson in November.
  
  Pyzchiva is approved to treat plaque psoriasis, active psoriatic arthritis, Crohn’s disease and ulcerative colitis in adults. In pediatric patients, the biosimilar is approved to treat moderate to severe plaque psoriasis and active psoriatic arthritis.
  
   
• Also on the biosimilar front, Formycon's biosimilar to Regeneron's blockbuster Eylea has gained an FDA nod.
  
  Branded as Ahzantive, the new biosim joins Formycon's Lucentis biosimilar on its roster of approved therapies. 
  
  The FDA approved Ahzantive to treat wet age-related macular degeneration, diabetic macular edema, diabetic retinopathy and macular edema following retinal vein occlusion.
  
  Formycon did not detail its launch plans in its approval press release.
   
• The European Commission has approved Valneva's Ixchiq as a single-dose vaccine to protect against chikungunya virus.
  
  Ixchiq is the world's first chikungunya vaccine, having gained prior approvals in the U.S. and Canada. Other regulatory reviews are progressing in the U.K. and Brazil, Valneva said in a press release.
  
  "In recent years, climate change has caused the Aedes mosquito, a known carrier of chikungunya and dengue viruses, to spread to areas in Europe that were previously unaffected," Valneva's chief medical officer, Juan Carlos Jaramillo, M.D., said in a statement.
  
  "It is critical to provide a vaccine solution not only to European travelers going to endemic chikungunya areas, such as South America or Africa, but also to the local European populations experiencing invasive mosquito attacks."