Welcome to 2024's regulatory tracker for Fierce Pharma.
On this page, we're recording the regulatory progress of in-market products, including expansions into key geographies and new indications. Some of these updates may not make the bar for standalone stories, but we think they are still worth mentioning.
UPDATED: Monday, April 22 at 11:35 a.m. ET
- Following an FDA approval earlier this month, Johnson & Johnson and Legend Biotech’s Carvykti nabbed a new approval from the European Commission in patients with relapsed and refractory multiple myeloma in patients who have received at least one prior line of therapy.
The approval marks the first BMCA CAR-T therapy authorized in EU to treat eligible patients as early as first relapse, according to a company press release (PDF). Most patients with the disease relapse after standard treatment and each following line of therapy is linked with lower response rates, which points to the necessity of new therapies earlier in the treatment pathway.
- UCB’s blockbuster hopeful Bimzelx is expanding its reach with a world-first European approval in moderate to severe hidradenitis suppurativa (HS). The nod marks the drug’s fourth European indication, joining approvals in plaque psoriasis, psoriatic arthritis and axial spondyloarthitis.
The latest indication was based on two phase 3 studies that showed a significantly higher proportion of Bimzelx-treated patients reaching a 50% or greater improvement in HS signs and symptoms by week 16.
Bimzelx is “the first and only approved medicine targeting IL-17A and IL-17F to the hidradenitis suppurativa community,” Emmanual Caeymaex, head of immunology solutions and U.S. at UCB, said in a release.
- England’s cost-effectiveness watchdog the National Institute for Heath and Care Excellence (NICE) recommended Menarini’s Stemline subsidiary’s Nexpovio as a combination treatment for multiple myeloma patients who have received one to two prior treatments.
Specifically, the drug was recommended alongside bortezomib and dexamethasone in patients who have had one previous line of treatment and don’t respond to Johnson & Johnson’s Darzalex and Bristol Myers Squibb’s Revlimid or have had two prior lines of treatment and don’t adequately respond to Revlimid.
NICE’s final draft guidance follows a previous recommendation for the med in combination with dexamethasone in specific myeloma patients.
Nexpovio is marketed by Karyopharm Therapeutics as Xpovio in the U.S.
- Also in Europe, Pfizer’s Emblaveo scored marketing authorization to treat patients with certain Gram-negative bacterial infections after an accelerated review.
The indication specifically covers complicated intra-abdominal infections (cIAI), hospital-acquired pneumonia (HAP), including ventilator-associated pneumonia (VAP), and complicated urinary tract infections (cUTI), including pyelonephritis.
The European Medicine Agency’s fast-tracked review “reflects the urgent need for new treatments to address the threat of antimicrobial resistance,” Pfizer’s chief international officer Aexandre de Germay said in a release.
Antimicrobial resistance is a major global concern with groups such as the World Health Organization marking it a critical priority.
- Sandoz also got a European approval for its biosimilar to J&J’s Stelara, Pyzchiva.
Samsung Bioepis-developed Pyzchiva is one of the first Stelara biosims to score marketing authorization in Europe and is a “crucial step towards offering European patients an additional safe and effective treatment option,” president of Sandoz Europe Rebecca Gutern said in a company statement.
The med also represents a major “value driver” for Sandoz and a key step in advancing the company’s growth strategy. The Novartis spin-out has commercialization rights to the Samsung med in the U.S., Canada, Europe, Switzerland and the U.K.
- Meanwhile in Canada, regulators granted Merck’s Prevymis a new indication as a prevention for cytomegalovirus disease in high-risk adult kidney transplant recipients.
The drug scored the same nod from the FDA last June after a speedy priority review. Both approvals were based on a phase 3 study of 589 kidney transplant recipients who received the drug orally or intravenously. The findings proved Prevymis to be non-inferior to Genentech’s Valcyte, the current standard-of-care, through 52 weeks post kidney transplant.
Prevymis was originally approved in Canada and the U.S. in 2017.
UPDATED: Friday, April 19 at 11:15 a.m. ET
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Takeda’s blockbuster Entyvio has racked up a new indication in its subcutaneous formulation. The FDA approved the subcutaneous form as a maintenance therapy for patients with moderately to severely active Crohn’s disease after prior treatment with intravenous Entyvio.
The nod follows the subcutaneous version’s September approval for ulcerative colitis maintenance treatment and is based on a phase 3 study of 409 patients. In the study, a statistically significant proportion of patients experienced long-term clinical remission on the newer form.
Patients can administer the treatment with a single-dose prefilled Entyvio pen “at home or on the go,” which serves to “demonstrate Takeda’s commitment to meeting the very real needs of those living with gastrointestinal diseases,” head of the company’s U.S. gastroenterology business unit, Brandon Monk, said in a release.
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Alvotech and Teva scored FDA approval for their biosimilar version of Johnson & Johnson’s immunology blockbuster Stelara, paving the way for a February 2025 launch.
The biosimilar partners dubbed their version Selarsdi and manufactured the med using the same type of cells and process used for Stelara production, the companies said in a release. Through a settlement with J&J, the drug can hit the market on February 21, 2025.
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Elsewhere, Alvotech inked an agreement with an unnamed strategic partner to boost U.S. access to its Teva-partnered high-concentration interchangeable Humira biosimilar. The move will broaden patient access and “further commitment to availability of vital biologics in the U.S. and globally,” Alvotech said.
Teva agreed to the partnership and will continue to commercialize the med under the Simlandi brand in the U.S.
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WuXi AppTec has secured a new regulatory clearance for a peptide facility in Changzhou, China.
The company’s site is a part of its ongoing expansion plan and has now received approval to produce meds from Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) following a three-day inspection.
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With a new complete response letter, the FDA has slapped down a bid for a ketamine treatment proposed by PharmaTher.
The agency initially granted the company a decision date of April 29 and in February outlined possible quality deficiencies it had noticed during the review.
PharmaTher says it has completed the agency's requested tests and is now confirming that its responses “accurately reflect the FDA’s comments,” CEO Fabio Chianelli said in a statement. “I believe that our goal to receive FDA approval for ketamine is closer.”
The company looks to solve the ongoing U.S. ketamine shortage and will seek international approvals to meet the “global demand” following FDA approval.
UPDATED: Tuesday, April 16 at 10:15 a.m. ET
- GSK's 5-in-1 meningococcal ABCWY vaccine candidate has been accepted for review by the FDA, the drugmaker said Tuesday.
The vaccine combines components of two existing vaccines, Menveo and Bexsero, both which feature "demonstrated efficacy and safety profiles," GSK said in its release.
Under the standard 10-month review timeline, GSK expects an FDA decision on the filing by February 14, 2025.
The company is chasing rival Pfizer, which won its own 5-in-1 meningococcal vaccine FDA approval last year. Pfizer's offering is branded as Penbraya.
- Novartis' cell therapy Kymriah has picked up a final draft recommendation from England's National Institute for Health and Care Excellence (NICE) for the treatment of patients up to 25 years old with B-cell acute lymphoblastic leukaemia (ALL) that is refractory, in relapse after a transplant or in second or later relapse.
In making the recommendation, NICE reviewed long-term follow-up results from three studies that supported the drug's 2018 approval, plus data from its use in England's Cancer Drugs Fund (CDF).
The drug has already reached 133 patients through the CDF and the recent recommendation "provides the opportunity for it to continue being a treatment option for many more," Novartis' managing director in the U.K. and Ireland, Andrée Gamache, said in a statement.
- Meanwhile, the United Kingdom's Medicines and Healthcare products Regulatory Agency (MHRA) has endorsed Takeda's Takhzyro to prevent attacks of hereditary angioedema (HAE) in patients ages 2 to 12, PMLiVE reports.
Previously, the drug was approved in this use for patients who are older than 12, according to the publication. Takhzyro is now the first option in the U.K. to prevent HAE attacks in children ages 2 to 6.
In a statement, HAE UK chief executive Angela Metcalfe called the approval a "huge milestone for the HAE community."
UPDATED: Thursday, April 11 at 9:25 a.m. ET
- AstraZeneca's Fasenra has secured a wider FDA approval to treat children ages 6 to 11 with severe asthma as an add-on maintenance treatment.
The drug was originally approved in 2017 as an add-on maintenance therapy for patients with severe eosinophilic asthma who are 12 and older.
For the latest approval, the FDA looked at data from various sources, including findings from an open-label phase 3 trial called TATE. In TATE, Fasenra delivered expected pharmacokinetic, pharmacodynamic and safety data in the 6- to 11-year-old patient group, AZ said in a release.
"We welcome additional treatment options for children living with severe asthma, a condition that remains complicated to manage, further helping to address the unmet need in this patient population and reducing the burden of disease for the broader asthma community," Lynda Mitchell, CEO of the Asthma & Allergy Network, said in AZ's release.
- In England, the National Institute for Health and Care Excellence (NICE) has endorsed AbbVie's Aquipta as a preventative option for patients who suffer from chronic and episodic migraines. The drug bears the brand name Qulipta in the U.S.
Specifically, NICE recommends the drug for patients who have had at least four migraine days per month and who've tried at least three other preventative treatment options.
Before the nod, the most effective treatments for these patients had to be injected. AbbVie's Aquipta presents a tablet alternative and may be preferred by some patients, Helen Knight, NICE's director of medicines evaluation, explained in the agency's release.
The NICE endorsement will open the therapy to about 170,000 patients in England and Wales.
UPDATED: Tuesday, April 9 at 10:45 a.m. ET
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ViiV Healthcare’s HIV treatment Dovato can now treat adolescents 12 years old and up with a new FDA nod.
The label expansion represents the first time an oral, two-drug single-tablet treatment regimen is available for the 12-year-old to 18-year-old patient population, ViiV said in a press release. In 2020, patients ages 13 to 24 made up 20% of new HIV diagnoses in the U.S., according to the company.
Dovato pairs integrase strand transfer inhibitor (INSTI) dolutegravir with nucleoside reverse transcriptase inhibitor (NRTI) lamivudine to inhibit the viral life cycle.
The latest approval was based on the company’s ongoing phase 3b DANCE study, which tested the drug in treatment-naïve adolescence and demonstrated that 26 out of 30 patients met and maintained viral suppression by week 48. -
Gilead Science’s COVID-19 treatment Veklury (remdesivir) scored an endorsement from England’s National Institute of Health and Care Excellence (NICE) in high-risk patients.
The thumbs-up was published as a final draft guidance, which noted that the cost-effectiveness estimates for the antiviral are within what NICE considers an acceptable use of the National Health Service (NHS)’s resources for hospitalized adults at a higher risk of serious illness.
Veklury was the first COVID treatment to win backing from Europe’s Committee for Medicinal Products for Human Use (CHMP) back in 2020 and later received a conditional marketing authorization from the European Medicines Agency, which converted the authorization to a full nod in 2022.
UPDATED: Friday, April 5 at 8:55 a.m. ET
- As part of UCB's bid to expand IL-17A and IL-17F inhibitor Bimzelx, the company said the FDA has accepted two new regulatory filings.
First, the FDA has accepted UCB's Biologics License Application (BLA) for Bimzelx to treat adults with moderate-to-severe hidradenitis suppurativa.
In addition, the agency has accepted the company's filing for a 2mL device presentations "to provide more options to optimize the individual patient experience," UCB said in a release. The drug is currently approved in 1mL presentations.
Besides those two filings, UCB has submitted Bimzelx applications in psoriatic arthritis, non-radiographic axial spondyloarthritis and ankylosing spondylitis.
UCB's drug won its initial FDA nod in October as a treatment for plaque psoriasis.
UPDATED: Wednesday, April 3 at 9:35 a.m. ET
- Bristol Myers Squibb's Reblozyl scored an approval from the European Commission as a first-line treatment for adults with transfusion-dependent anemia due to very low, low and intermediate-risk myelodysplastic syndromes (MDS).
European officials based the decision on results from the phase 3 COMMANDS study. In the trial, Reblozyl bested the popular erythropoiesis stimulating agent, epoetin alfa, on the primary endpoint of concurrent red blood cell transfusion independence and hemoglobin increase.
The approval means that more patients in Europe will "have the potential to become transfusion independent for longer periods of time compared to current options available,” BMS' head of European markets, Monica Shaw, M.D., said in a statement.
Reblozyl is already approved in the U.S. and Japan for the first-line treatment of anemia associated with MDS.
- In China, Hangzhou Jiuyuan Gene Engineering is planning a regulatory filing for a biosimiliar of Novo Nordisk's diabetes drug Ozempic, Reuters reports.
It remains unclear when Hangzhou Jiuyuan Gene Engineering might be able to launch its proposed biosimilar. Novo's Ozempic patent in China runs until 2026, Reuters reports, citing the company's annual report.
- Spain's Rovi has picked up an FDA approval for its long-acting schizophrenia treatment Risvan.
Risvan is a prolonged-release injectable version of the decades-old antipsychotic drug risperidone. Rovi says its treatment offers "immediate and sustained plasmatic drug levels" as of the first injection.
Analysts with ODDO BHF expect the company to seek a partner to help with the U.S. launch.
UPDATED: Thursday, March 28 at 11:20 a.m. ET
- After a fast FDA approval in October, the European Commission has signed off on Merck's Keytruda as a continuous treatment used both before and after surgery to treat resectable non-small cell lung cancer (NSCLC) at high risk of recurrence.
The approval was based on an overall survival win from the Keynote-671 trial, in which perioperative Keytruda, paired with presurgical chemotherapy, slashed the risk of death by 28% in patients with resectable stage 2 to 3b NSCLC compared with chemo alone. The Keytruda-containing regimen also slashed the risk or disease recurrence, progression or death by 41%.
- Biocon and its partner Zentiva have won an approval in the U.K. for their drug-device copycat of Novo Nordisk's GLP-1 diabetes drug Victoza.
Biocon boasts several firsts in the past with its biosimilars, including Semglee, a copycat to Sanofi's Lantus and the first interchangeable biosimilar of an insulin product.
The new U.K. approval is a testament to Biocon's "strong focus on GLPs and peptides as important future growth drivers for the generics business," Siddharth Mittal, CEO and managing director of Biocon, said in a statement.
- The FDA has expanded Gilead Sciences' antiviral Vemlidy to a younger patient population. The new nod allows the tenofovir product to be given 25 mg once-daily for chronic hepatitis B infection in patients as young as 6 years old and weighing at least 25 kg with compensated liver disease, extending a 2022 nod that allows the drug to reach patients 12 years of age and above.
The approval was based on 96-week viral suppression results from a phase 2 trial, including 18 patients aged 6 to less than 12 years.
UPDATED: Wednesday, March 27 at 11:18 a.m. ET
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AstraZeneca and Sanofi’s respiratory syncytial virus (RSV) antibody, Beyfortus, will be able to protect babies in Japan next RSV season after scoring approval from the country’s Ministry of Health, Labour and Welfare.
The nod covers the prevention of lower respiratory tract disease (LRTD) in all newborns, infants and children entering their first or second RSV season. More than 100,000 cases of RSV LRTD in infants are reported each year in Japan, AstraZeneca said in a company release.
“The approval of Beyfortus means it is possible for the first time to prevent serious respiratory disease due to RSV for all infants in Japan and reduce the infectious disease burden on healthcare systems,” AZ’s executive vice president of vaccines and immune therapies, Iskra Reic, added in the release.
Beyfortus is expected to be available for use in the country for the upcoming 2024/2025 RSV season.
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After a nod from the European Commission, Otsuka and Lundbeck’s blockbuster antipsychotic franchise, Abilify, is reaching new heights with the introduction of a long-acting injectable formulation.
Called Abilify Maintena, the intramuscular injection is approved for use in schizophrenia patients as an every-other-month maintenance treatment. It’s Europe's first long-acting antipsychotic injectable to hit the market with that dosing schedule.
“This treatment aims to increase patient adherence and convenience, contributing to the careful and comprehensive management of this chronic condition,” Lundbeck’s head of R&D Johan Luthman, M.D., Ph.D., noted in a press release.
The new option could also “allow patients and their healthcare practitioners to focus on other elements of care,” head of medical affairs at Otsuka Europe Peter Gillberg, M.D., added.
UPDATED: Tuesday, March 26 at 10:15 a.m. ET
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Astellas’ gastric cancer med Vyloy secured a world-first approval from Japan’s Ministry of Health, Labour and Welfare. The approval represents the first global nod for an anti-claudin 18.2 (CLDN18.2)-targeted therapy.
In Japan, the monoclonal antibody was approved to treat CLDN18.2 positive, unresectable advanced or recurrent gastric cancer.
Vyloy marks the dawn of a “new era” in the treatment of gastric cancer, Astellas’ head of immuno-oncology development, Moitreyee Chatterjee-Kishore, Ph.D., said in a company press release. Gastric cancer is the third-deadliest cancer in Japan, according to Astellas.
The approval was based on results from the company’s phase 3 SPOTLIGHT and GLOW studies, which tested the drug as a first-line treatment in the patient group along with two different combination chemotherapy regimens. In both studies, the drug showed statistical significance in progression-free survival and overall survival compared to placebo and chemotherapy.
Astellas has submitted approval bids with regulatory agencies across the globe.
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Takeda also scored a nod in Japan for its intravenous Adzynma injection, clearing the med to treat ultra-rare chronic blood clotting disorder congenital thrombotic thrombocytopenic purpura (cTTP).
Adzynma is the only approved recombinant ADAMTS13 therapy that can replace the deficient ADAMTS13 enzyme in patients with cTTP, which causes the disease.
cTTP patients “had limited treatment options and now have the first treatment option specifically approved to treat this ultra-rare condition,” Takeda’s Japan region R&D head Yasushi Kajii said in a recent release.
The approval was based on a phase 3 study of five Japanese patients. An interim analysis showed that no patient in the trial experienced an acute TTP event while taking Adzynma, while one patient did have such an event while receiving plasma-based therapies during the study’s comparison period.
The med was approved by the FDA in November and was granted orphan drug designation by regulators in the U.S., Europe and Japan.
UPDATED: Thursday, March 21 at 1:32 p.m. ET
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Bristol Myers Squibb’s CAR-T drug Abecma can now reach a wider group of multiple myeloma patients across the European Union's member states thanks to an expanded approval from European regulators.
The European Commission granted approval for Abecma to treat patients with relapsed and refractory multiple myeloma who have progressed after treatment with two prior therapies. Prior treatment regimens could include an immunomodulatory agent, a proteasome inhibitor or an anti-CD38 antibody.
Abecma gained approval as a fourth-line treatment in Europe in 2021.
The latest approval is an “exciting milestone,” the company’s head of European markets, Monica Shaw, M.D., said in a release.
BMS says it's ready to meet the extra demand by introducing a “significant increase” in manufacturing capacity. The company looks to make the med available in the new indication following completion of reimbursement talks with individual countries.
UPDATED: Tuesday, March 12 at 11:20 a.m. ET
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Johnson & Johnson is off to the races with its next potential Tremfya indication.
Monday, the company said it submitted an FDA application for Tremfya (guselkumab) to treat adults with moderately to severely active ulcerative colitis (UC). The application hinges on J&J’s phase 3 Quasar program, which is testing Tremfya in people living with moderately to severely active UC who don’t respond to or are intolerant to standard therapy, prior biologics and/or JAK inhibitors.
Tremfya first passed muster with U.S. regulators in July 2017, when it snagged a green light to treat patients with moderate-to-severe plaque psoriasis. In 2020, the drug was approved to treat adults with active psoriatic arthritis.
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Across the pond, the United Kingdom’s Healthcare products Regulatory Agency (MHRA) approved Daiichi Sankyo’s acute myeloid leukemia (AML) drug Vanflyta, also known by its generic moniker quizartinib. Each year, some 3,100 people are diagnosed with AML in the U.K., which can lead to bleeding, anemia and infections, the MHRA explained in a release.
Daiichi’s drug is taken orally once per day alongside chemotherapy as a first-line treatment for adults who have a certain mutation in the FLT3 gene. Vanflyta works by blocking the action of the proteins known as tyrosine kinases, which can cause the body to produce large numbers of abnormal bone marrow cells that don’t mature to become healthy cells.
In the phase 3 trial Daiichi used to secure its approval, patients on Vanflyta had a median overall survival of 31.9 months, versus just 15.1 months in the control cohort.
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Meanwhile, the U.K. has also approved Pfizer’s long-term UC drug Velsipity, also known as etrasimod.
Velsipity works by preventing lymphocytes—a type of white blood cell—from travelling through the lymph nodes into the blood. Those lymphocytes are implicated in the immune response and inflammation that is linked to the development of UC, the MHRA said in a release.
The MHRA based its approval on results from two clinical trials—Elevate UC 52 and Elevate UC 12—which together enrolled nearly 750 patients ages 16 and up for whom standard UC treatment or other treatments did not work well enough.
The combined study results showed that 26% of patients who received Velsipity achieved clinical remission, versus just 11% in the placebo group, at the 12-week mark.
UPDATED: Monday, March 11 at 10:00 a.m. ET
- Regeneron's PCSK9 inhibitor Praluent won an FDA approval to treat children 8 and older with heterozygous familial hypercholesterolemia (HeFH), the company said in a Monday release.
HeFH is a form of familial hypercholesterolemia (FH), an inherited condition that causes patients to develop dangerous levels of bad cholesterol. Previously, Praluent won FDA approval to treat adults with HeFH in 2015.
From a regulatory perspective, the new approval allows Praluent to catch up to Regeneron's in-class rival Repatha, which won a pediatric HeFH nod in 2021.
Besides FH, Regeneron's drug is approved to treat clinical atherosclerotic cardiovascular disease. Both approvals are for add-on uses alongside statins, diet and exercise.
- Travere Therapeutics has submitted a supplemental new drug application (sNDA) to the FDA in pursuit of a full approval for Filspari in IgA nephropathy.
The drug won its accelerated FDA approval about a year ago in the rare kidney disease.
The company's sNDA filing is based on 2-year results from the phase 3 PROTECT confirmatory study. In the study, Filspari failed to reach statistical significance over the control drug irbesartan when it came to kidney function outcomes as measured by the estimated glomerular filtration rate (eGFR).
Despite the trial miss, Filspari is touting a reduction in proteinuria, or protein in the urine, and long-term preservation of kidney function seen in the study.
The FDA has 60 days to decide whether to accept the company's application. Just two weeks ago, the drug won support from regulators in Europe.
UPDATED: Friday, March 8 at 10:15 a.m. ET
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Pfizer-BioNTech’s and Moderna’s mRNA-based COVID-19 vaccines have been cleared of a specific side effect concern in Europe.
On Friday, the European Medicines Agency’s (EMA’s) safety committee, PRAC, found “insufficient evidence” linking Pfizer and BioNTech’s Comirnaty and Moderna’s Spikevax to cases of postmenopausal bleeding. As a result, an update to the vaccines’ product information is “not warranted,” PRAC concluded.
Postmenopausal bleeding is vaginal bleeding that occurs one year or more after a person’s last menstrual period. It is “always considered abnormal” and can point to serious medical conditions, EMA said.
PRAC started its investigation after medical literature and post-authorization data suggested a potential link. The safety committee ultimately determined that the available data “do not support a causal association.”
UPDATED: Wednesday, March 6 at 9:30 a.m. ET
- GSK's PD-1 cancer drug, Jemperli, has gained a recommendation from England's National Institute for Health and Care Excellence (NICE) to treat patients with advanced or recurrent mismatch repair deficient (dMMR) or microsatellite instability-high (MSI-H) endometrial cancer.
In a release, NHS England said an estimated 150 to 200 patients per year will be eligible for the therapy.
Nearly two thirds of patients treated with Jemperli and chemotherapy did not see cancer progression after 12 months in clinical trials, according to NHS England. That compared with 24% for chemotherapy alone.
- Also at NICE, Sanofi's rare disease drug Xenpozyme failed to secure a recommendation to treat certain patients with acid sphingomyelinase deficiency, also known as Niemann-Pick Disease.
In a statement, Sanofi's country lead for the U.K. and Ireland, Rippon Ubhi, said the company is "extremely disappointed and saddened by the decision," describing it as "devastating news" for patients.
The progressive disease affects about 40 people in the U.K., according to the drugmaker. Xenpozyme is the only licensed drug for the condition.
Officials in Scotland have already enabled access to the therapy, Sanofi said, so the decision creates "inequity in access" within the U.K., Ubhi said.
"In our view, patients with ultra-rare diseases in England and Wales are being let down by an inflexible appraisal system which fails to recognise the benefits and full value of treatments such as olipudase alfa for patients, carers, families and wider society," Ubhi continued.
UPDATED: Tuesday, March 5 at 10:30 a.m. ET
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England's cost watchdogs at the National Institute for Health and Care Excellence (NICE) opted to not recommended AstraZeneca and Daiichi Sankyo’s Enhertu in certain patients over pricing concerns.
Specifically, Enhertu is not recommended for NHS coverage in the treatment of HER2-low metastatic or unresectable breast cancer after chemotherapy. NICE issued a similar draft guidance in September before engaging in the negotiations.The drug is priced at £1,455 per vial, minus an undisclosed discount. The list price fell above the range that NICE considers an acceptable use of NHS resources, the agency said in an emailed press release.
The NHS and the drugmakers have been attempting to agree on an acceptable price since NICE paused its appraisal process in December, but the negotiations fell flat.
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AbbVie’s dermal filler Juvederm Voluma XC expanded its reach with a new FDA indication to improve moderate to severe temple hollowing through injection in the temple region.
The approval marks the first for a hyaluronic acid dermal filler’s use in the upper face, president of AbbVie’s Allergan Aesthetics, Carrie Strom, said in the company’s press release.
In a clinical trial, 80% of participants saw at least a one-point improvement in temple hollowing three months after treatment; 73% saw the effects last more than one year
UPDATED: Friday, March 1 at 11:15 a.m. ET
- England's National Institute for Health and Care Excellence (NICE) has recommended Johnson & Johnson's Darzalex, plus three other drugs, as a combination treatment for newly diagnosed systemic amyloid light-chain (AL) amyloidosis.
Also included in the combo regimen are the proteasome inhibitor bortezomib, the chemotherapy cyclophosphamide and the steroid dexamethasone. The combo, known as DVCd, is the first licensed treatment for people with the life-threatening disease, according to a release from J&J.
Around 600 people in the United Kingdom suffer from AL amyloidosis each year, according to the drugmaker.
NICE originally recommended against usage of the combo in November 2022, but an appeal has now yielded the agency's recommendation.
DVCd has been available in Scotland since 2022, according to a release from Myeloma UK.
"Although we’re immensely proud of what we’ve achieved together, we are mindful that, over the last year, while we pushed for a U-turn on NICE’s decision, hundreds of newly-diagnosed patients missed out on this life-changing treatment—a treatment which has been available in Scotland since 2022," Myeloma UK chief executive Sophie Castell, Ph.D., said in a statement. "It’s disappointing that it took so long to get to this point."
- Meanwhile, the United Kingdom's Medicines and Healthcare products Regulatory Agency (MHRA) approved Amgen's prefilled syringe version of Xgeva.
The drug is used to prevent serious complications from bone metastasis and to treat giant cell tumors of the bone, according to the agency.
The prefilled syringe version of the drug was approved after only a 30-day review under a new pathway that allows the MHRA to take "into account the expertise and decision-making of trusted regulatory partners in the authorisation process."
Amgen's prefilled syringe version of Xgeva is the first drug approved under the pathway, which is called the International Recognition Procedure (IRP).
UPDATED: Thursday, Feb. 29 at 9:10 a.m. ET
- Takeda is working with officials in India to make the company's dengue vaccine shot, Qdenga, available in the country, an executive told Reuters.
Gary Dubin, Takeda's vaccines head, told the news service that the company is engaging with regulators and plans "to start a clinical trial very soon."
So far, the company has launched Qdenga in Indonesia, Thailand, Brazil and Argentina, plus certain European markets.
Takeda this week tapped India's Biological E to help ramp up production of the shot.
- Also in India, Eli Lilly expects to launch its new GLP-1 diabetes and obesity drug Mounjaro as soon as next year, Reuters reports, citing an interview with CEO David Ricks.
The drug is under review in India, the CEO told Reuters.
Speaking at a conference in the country, the Big Pharma CEO said that India has some "redundant" policies that delay the introduction of new medicines there, according to India's Business Today.
- Elsewhere in Asia, South Korea has granted approval to Sanofi and Regeneron's Dupixent to treat moderate to severe prurigo nodularis for patients who've been unsuccessful on other therapies, the Korea Biomedical Review reports.
In Korea, Dupixent also carries approvals to treat atopic dermatitis and severe eosinophilic asthma, according to the publication.
UPDATED: Tuesday, Feb. 27 at 9:20 a.m. ET
- After buying Global Blood Therapeutics and its sickle cell disease drug Oxbryta in 2022, Pfizer is having trouble winning a coverage recommendation in England.
Tuesday, England's National Institute for Health and Care Excellence (NICE) recommended against (PDF) usage of the drug within its marketing authorization. Specifically, NICE recommended against using Oxbryta for treating hemolytic anemia caused by sickle cell disease in people 12 years and over.
NICE has "outstanding uncertainties" about the company's application and wants more information from Pfizer.
Around 4,000 people would be eligible for the treatment, if it were recommended, according to NICE.
- AbbVie's FDA application for Epkinly in difficult-to-treat relapsed or refractory follicular lymphoma has picked up a priority review.
The company's filing leverages the phase 1/2 EPCORE study, which showed strong and durable treatment responses in third-line patients with relapsed or refractory follicular lymphoma, according to the drugmaker.
Under the FDA's priority review program, the agency's review period for this application will be 6 months rather than the usual 10.
- Meanwhile, the FDA has accepted BeiGene's application for Tevimbra to treat first-line gastric or gastroesophageal junction cancers.
The agency set a target decision date in December for the company's filing.
BeiGene's application leverages data from the RATIONALE-305 trial, which met its primary endpoint of overall survival when comparing Tevimbra plus chemotherapy against placebo and chemotherapy.
The Tevimbra arm was associated with a higher objective response rate and duration of response, as well.
- Theratechnologies' FDA filing for an intramuscular maintenance dose of the HIV med Trogarzo has been hit with a Refusal to File letter.
After a preliminary review of the company's recent application, the FDA found that the filing "was not sufficiently complete to permit a substantive review," Theratechnologies said in a release.
The company is now reviewing its options.
UPDATED: Thursday, Feb. 22 at 4:50 p.m. ET
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Johnson & Johnson’s expansion push for its Rybrevant will get a quick review from the FDA.
The agency accepted the drugmaker's submission for its combination of Rybrevant and Yuhan-partnered lazertinib for first-line treatment of locally advanced or metastatic EGFR-mutated non-small cell lung cancer (NSCLC) with a priority review designation, J&J announced in a social media update.
J&J’s bid is based on its phase 3 MARIPOSA trial, which pit Rybrevant and lazertinib against AstraZeneca’s Tagrisso in a high-stakes head-to-head study. In the trial, the Rybrevant combo reduced the risk of disease progression or death by 30% compared with Tagrisso and improved the median time patients lived without their disease getting worse by 7.1 months to reach 23.7 months.
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Pfizer’s alopecia med Litfulo was recommended by the U.K.’s National Institute for Health and Care Excellence (NICE) for coverage on the region’s National Health Service (NHS), making it the first treatment for alopecia areata to score a NICE nod.
The drug, a JAK inhibitor, was authorized in November by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) after bagging its FDA approval in June.
Both regulatory agencies based their decision on the company’s phase 2b/3 ALLEGRO study, which proved that 23% of patients who were suffering from 50% or more scalp hair loss achieved 80% or more scalp hair coverage after six months compared to placebo’s 1.6%. -
GSK also received a NICE recommendation for its Omjjara as a treatment option for disease-related splenomegaly.
The endorsement is the latest in the med’s regulatory winning streak. Earlier this month, the JAK inhibitor nabbed an authorization from the MHRA as a treatment for myelofibrosis in patients who have moderate to severe anemia.
Omjjara, which is branded in the U.S. as Ojjaara, won its FDA approval in September for myelofibrosis in anemia patients. GSK has previously said that the drug will be able to bring in peak sales above $1 billion.
UPDATED: Wednesday, Feb. 21 at 3:20 p.m. ET
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Novartis’ sickle cell disease treatment Adakveo will no longer be available in the U.K. as the company opted to pull it from the market.
The recall comes after the Medicines and Healthcare products Regulatory Agency (MHRA) revoked the drug's conditional marketing authorization, citing an unfavorable benefit-risk balance, which followed a similar move by Europe’s health authorities in May.
The med is meant to reduce the frequency of vasco-occlusive crises, a common complication of sickle cell disease. In a phase 3 study, Adakveo didn’t show an improvement over placebo. As it stands, the drug is still sold in the U.S. market following its 2019 FDA approval.
Novartis recently noted that it “continues to discuss” the study results with the FDA and other global health authorities. -
The FDA signed off on a new reduced dosing frequency for Johnson & Johnson’s BCMA-targeted bispecific drug Tecvayli.
Now, certain patients with relapsed or refractory multiple myeloma (RRMM) can take the drug every two weeks. Specifically, patients must have achieved and maintained a complete response or better for at least six months.
Tecvayli was approved by the FDA in 2022 as a RRMM treatment for patients who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 antibody. The drug’s European label is less limited and requires three prior therapies.
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Harmony Biosciences’ pediatric ambitions for its Wakix will get a speedy review from the FDA. The agency accepted the drugmaker’s label expansion filing under priority review and set a target decision date of June 21.
The company hopes to expand the excessive daytime sleepiness (EDS) med’s usage to cover patients with narcolepsy who are ages six years and older. EDS is a common characteristic of sleep disorder narcolepsy.
The European Medicines Agency last year granted pediatric approval based on the phase 3 study that the FDA will now review.
The drug won its original FDA approval in 2019, which made Wakix the only approved med for the condition that isn’t scheduled as a controlled substance.
UPDATED: Tuesday, Feb. 20 at 11:15 a.m. ET
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The FDA is looking over another indication for Merck’s superstar cancer med Keytruda, with a decision date now expected by June 21.
The agency accepted the company’s application to treat patients with primary advanced or recurrent endometrial carcinoma, along with chemotherapy, and granted a speedy review.
In Merck’s phase 3 NRG-GY018 study, which was sponsored by the National Cancer Institute, Keytruda beat solo chemotherapy at starving off tumor progression or death by 46% in patients whose cancer was mismatch repair proficient and by 70% in patients whose cancer was mismatch repair deficient.
If approved, the drug would be the first immunotherapy cleared for the frontline treatment of advanced endometrial cancer regardless of mismatch repair status, vice president of global clinical development at Merck Research Laboratories, Gursel Aktan, M.D., said in a company release.
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June 21 also serves as the FDA's target decision date for a new use of Bristol Myers Squibb’s recently acquired Krazati.
The company seeks a label expansion to treat patients with previously treated KRAS G12C-mutated locally advanced or metastatic colorectal cancer alongside chemotherapy cetuximab. BMS’ application was accepted under an FDA priority review.
Krazati just recently joined BMS’ arsenal after the company bought its maker, Mirati Therapuetics, for up to $5.8 billion in October. The non-small cell lung cancer med brought in $16.4 million for Mirati over last year’s third quarter before the buyout.
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Argenx, too, expects to hear regulatory news by June 21 as the FDA weighs a chronic inflammatory demyelinating polyneuropathy indication for Vyvgart Hytrulo.
The company used a priority review voucher to speed up the application as patients with the autoimmune disease have “long been waiting” for new treatment options, argenx’s chief medical officer, Luc Truyen, said in a company release.
In the largest trial to date in the disease, Vyvgart Hytrulo was associated with a 61% lower risk of relapse compared with placebo.
Analysts at ODDO BHF previously pegged Vyvgart peak sales to reach $13.6 billion by 2035.
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Following Pfizer's Velsipity FDA nod in October, the ulcerative colitis med has expanded its footprint with a European approval.
The European Commission signed off on Velsipity after the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended an approval in December. While the drug is approved for adults in the U.S., the European label covers patients 16 years of age and older.
Pfizer bought Velsipity through its $6.7 billion Arena Pharmaceuticals acquisition. The drug rivals Bristol Myers Squibb’s Zeposia.
UPDATED: Friday, Feb. 16 at 9:15 a.m. ET
- The FDA has converted an accelerated approval for Merck KGaA's first-in-class MET inhibitor Tepmetko into a full approval based on a longer follow up of tumor response data.
The drug's indication remains unchanged. Tepmetko won its original accelerated approval in 2021 to treat metastatic non-small cell lung cancer harboring MET exon 14 skipping alteration.
The conversion to traditional approval analyzed an additional 161 patients and added 28 months of follow-up time to assess the duration of response from the phase 2 VISION trial.
In the updated analysis, Tepmetko triggered a response in 57% of treatment-naïve patients and 45% of previously treated patients. Both numbers marked an improvement from the previously reported 43% overall response rate in a smaller patient group. The responses lasted for at least a year in 40% of responders in the first-line setting, or in 36% of responders among previously treated patients.
- Astellas said Japan's Ministry of Health, Labour and Welfare have granted priority review to its application for Padcev and Merck's Keytruda as first-line treatment for metastatic bladder cancer.
The FDA in December handed the combo a full approval in the indication based on the landmark overall survival showing from the EV-302 trial. The European Medicines Agency is also reviewing the regimen.
UPDATED: Wednesday, Feb. 14 at 4:05 p.m. ET
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After an initial approval in lung cancer in November, Bristol Myers Squibb’s Augtyro is officially under FDA evaluation for another use.
The FDA accepted BMS’ application for Augtyro in solid tumors with a neurotrophic tyrosine receptor kinase (NTRK) gene fusion that are locally advanced or metastatic or where surgery will likely result in severe morbidity. The agency tagged the application with priority review status and expects to make a decision by June 15.
BMS padded the application with study results showing the drug's ability to achieve “clinically meaningful response rates” and robust durability of response, the company said in its statement.
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Dermavant is chasing what CEO Todd Zavodnick previously called “blockbuster, game-changing status” for its steroid-free topical med Vtama. To get there, the company will have to lock down a label expansion to include atopic dermatitis, which it has now officially filed for with the FDA.
Currently, the cream is approved as a plaque psoriasis treatment. Sales are off to a bit of a slow start since its May approval, but an atopic dermatitis nod would open the treatment up to a much larger population. Dermavant, a Roivant subsidiary, based its application in that use on positive data from three phase 3 trials.
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After a July rejection, Citius Pharmaceuticals is once again seeking FDA approval for its Lymphir in relapsed or refractory cutaneous T-cell lymphoma (CTCL).
Upon its initial rejection, the FDA alerted the company that it would have to include enhanced product testing and “additional manufacturing controls” to pass muster, the company said in its release. The agency didn’t flag any issues with the safety and efficacy of the drug’s data package.
Now, Citius believes that it has addressed the issues with its first application and has thus resubmitted its drug for FDA approval. The company bought Lymphir for $40 million from Dr. Reddy’s in 2021.
UPDATED: Tuesday, Feb. 13 at 9:10 a.m. ET
- In Europe, Vertex's Casgevy has gained a conditional marketing authorization to treat certain patients with sickle cell disease and transfusion-dependent beta thalassemia (TDT).
In sickle cell disease, the CRISPR/Cas9 gene-edited therapy is authorized to treat patients 12 and older who have severe disease, which is characterized by recurrent vaso-occlusive crises.
In TDT, the drug is eligible to treat patients for whom hematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen matched related HSC donor is not available, Vertex said in a release.
Between the two indications, about 8,000 patients in Europe could be eligible for treatment, the company estimates.
Under Europe's conditional authorization system, approvals are valid for one year and regulators can re-review the available data to renew the nods as needed.
With the authorization now in place, Vertex can amplify its launch efforts in individual countries. The company is "already working closely with national health authorities to secure access for eligible patients as quickly as possible," Vertex said in its release.
UPDATED: Friday, Feb. 9 at 9:25 a.m. ET
- England's National Institute for Health and Care Excellence (NICE) has endorsed Stada and Calliditas Therapeutics Kinpeygo for certain adults with primary immunoglobulin A nephropathy (IgAN) when there is a risk of rapid disease progression. To be eligible, patients must have a urine protein-to-creatinine ratio of 1.5 g/g or more.
With the recommendation, Kinpeygo, or targeted-release budesonide, becomes the first-ever drug for primary IgA nephropathy to gain a NICE endorsement.
A Stada affiliate plans to make the drug available in the spring, the company said in a release.
"To now be able to treat my patients with a drug that can modify the fundamental cause of this important cause of kidney failure is a real game changer," Professor Jonathan Barrett, of the University of Leicester, said in a statement provided by Stada.
- Meanwhile, argenx's subcutaneous version of Vyvgart has picked up an approval from the United Kingdom's Medicines and Healthcare products Regulatory Agency (MHRA) to treat generalized Myasthenia Gravis.
With the latest nod, argenx's subcutaneous drug is approved as an add-on to standard therapy for adults with gMG who are anti-acetylcholine receptor (AChR) antibody positive.
The intravenous version of the drug had won an MHRA approval in March 2023.
"Our focus now will be on ensuring seamless access to this medicine for all those who could potentially benefit, and we are working closely with authorities across the UK to make this a reality," argenx UK general manager, David Knechtel, said in a statement.
- Takeda's dengue vaccine launch is gaining steam around the globe.
In Malaysia, the Drug Control Authority has granted a conditional approval to Qdenga to prevent dengue fever in people 4 and older.
Takeda will be required to provide regular updates on the efficacy and safety of the vaccine in the country.
The vaccine has already launched in Indonesia, Brazil, Thailand and Argentina, Takeda said in a recent corporate update (PDF). In private markets, the company said it has seen "strong initial demand."
UPDATED: Wednesday, Feb. 7 at 9:15 a.m. ET
- As GSK clashes with vaccine rival Pfizer on the RSV vaccine front, the British drugmaker is looking to expand its shot into a larger eligible population.
This week, the company's FDA application for Arexvy in people ages 50 to 59 with underlying medical conditions earned a priority review designation. Under the speedy review timeline, the FDA aims to decide on GSK's filing by June 7.
Officials in Europe are already reviewing potential use of the vaccine in people 50 to 59. There, GSK expects a decision in the second half of the year.
The news comes after GSK's shot generated 1.2 billion pounds sterling ($1.5 billion) during its first four months on the market, the company said in late January.
- Elsewhere, GSK is looking to expand the label of shingles vaccine Shingrix in China.
While the vaccine is already approved in people 50 and older in China, GSK is seeking an approval for Shingrix use in at-risk people who are 18 and older. Regulators in China have accepted the company's filing.
Supporting GSK's application are six trials in patients 18 and older who have undergone stem cell transplantation, kidney transplants, or who have cancer or HIV, the company said.
- After a positive readout in the CheckMate-77T trial, Bristol Myers Squibb is advancing its perioperative Opdivo ambitions.
Regulators in Europe and the U.S. accepted the company's application for Opdivo plus chemotherapy—followed by surgery and adjuvant Opdivo—in patients with resectable stage IIA to IIIB non-small cell lung cancer (NSCLC), the company said Wednesday.
The U.S. FDA is expected to act on the filing by October 8, BMS said.
Bristol's applications are based on results from CheckMate-77T. The trial showed that adding Opdivo before and after surgery significantly reduced the risk of tumor recurrence, progression or death in patients with early-stage NSCLC compared with chemo alone before surgery.
Besides BMS, oncology giants AstraZeneca and Merck have each trumpeted their own perioperative wins for their checkpoint inhibitors.
UPDATED: Friday, Feb. 2 at 8:45 a.m. ET
- After a prior preliminary rejection, AbbVie's Tepkinly has gained a usage recommendation (PDF) from England's National Institute for Health and Care Excellence (NICE).
NICE endorsed the drug, also known as epcoritamab, for adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who've tried at least 2 prior systemic treatments. To be eligible, patients must have already tried Roche's antibody-drug conjugate Polivy, or not be eligible for the drug.
In addition, AbbVie must provide Tepkinly under an agreed-upon "commercial arrangement," NICE said.
The latest decision follows a preliminary rejection for the drug in November.
Jefferies analysts have predicted AbbVie's Tepkinly can generate $2.75 billion at peak, pharmaphorum reports.
- GSK's Omjjara has extended its regulatory winning streak.
This week, the United Kingdom's Medicines and Healthcare products Regulatory Agency (MHRA) endorsed the drug to treat adults with myelofibrosis who have moderate to severe anemia.
The drug recently won its first European Commission approval, and GSK's filing is moving ahead in Canada, as well. In the U.S., the FDA approved Ojjaara last year.
UPDATED: Tuesday, Jan. 30 at 3:00 p.m. ET
- While one Abecma filing stalls at the FDA, Bristol Myers Squibb has received a pair of priority reviews for its other CAR-T drug, Breyanzi.
The FDA granted priority reviews to Bristol's applications for Breyanzi in previously treated follicular lymphoma and post-BTK mantle cell lymphoma. The agency's target decision dates are May 23 and May 31, respectively.
Meanwhile, another filing for Breyanzi in chronic lymphocytic leukemia or small lymphocytic lymphoma following a BTK inhibitor and BCL-2 inhibitor is progressing under a separate FDA priority review. That application carries a target action date of March 14.
- Eli Lilly has won approval in Canada for Olumiant as a treatment for severe alopecia areata. The drug is not recommended for use with other JAK inhibitors, biologic immunomodulators, azathioprine, cyclosporine or other potent immunosuppressants.
The Lilly JAK inhibitor beat placebo in two phase 3 trials at helping patients recover hair. Across the studies at 36 weeks, 17% to 22% of patients taking Olumiant at 2 mg/day and 32% to 35% of patients taking the 4 mg/day strength achieved 80% or more scalp hair coverage. Only 3% to 5% individuals taking placebo could say that.
Back in June 2022, Olumiant became the first drug specifically approved by the U.S. FDA to treat alopecia.
- Following an FDA approval in September and a European Commission nod Monday, GSK's Ojjaara, or momelotinib, has moved closer to a go-ahead in Canada. Local health authorities have accepted GSK's application for the JAK inhibitor as a myelofibrosis treatment.
An estimated 1,400 to 2,177 patients are living with myelofibrosis in Canada, according to GSK. Globally, about 82% of myelofibrosis patients are diagnosed with intermediate or high-risk disease, and about one-third to one-half of patients are anemic at the time of diagnosis.
UPDATED: Monday, Jan. 29 at 1:15 p.m. ET
- Partners AstraZeneca and Daiichi Sankyo announced that a label expansion filing for their antibody-drug conjugate Enhertu has gained an FDA priority review.
The filing seeks FDA approval for the drug to treat certain patients with unresectable or metastatic HER2-positive solid tumors who have received prior treatment or who have no satisfactory alternative treatments.
The partners' tumor-agnostic application leverages data from the DESTINY-PanTumor02 trial, which showed that Enhertu was associated with "clinically meaningful and durable responses" in patients with various solid tumor types, according to the companies.
AZ and Daiichi Sankyo expect the FDA's decision on their filing in the second quarter of 2024.
- Drugs in Takeda's immune globulin franchise have gained expanded approvals on opposite sides of the Atlantic.
In the U.S., the FDA approved Gammagard Liquid to improve neuromuscular disability and impairment in adults with chronic inflammatory demyelinating polyneuropathy (CIDP). The drug is an immune globulin infusion that comes without the human enzyme hyaluronidase. Under the new label, Gammagard Liquid can be used as an induction therapy, Takeda said in a Monday release.
The expanded FDA nod follows a recent approval for Takeda's Hyqvia, an immune globulin infusion with hyaluronidase, as a CIDP maintenance therapy for U.S. patients.
“Together with the recent Hyqvia approval in the U.S., we can now offer induction and maintenance therapy options to adults living with CIDP that may accommodate their personal treatment needs," Richard Ascroft, head of Takeda’s U.S. plasma-derived therapies unit, said in a statement.
As for Hyqvia, that treatment just gained European Commission approval as a maintenance therapy to treat patients with CIDP after stabilization with intravenous immunoglobulin therapy (IVIG), Takeda said Monday.
- Also in Europe, GSK's Omjjara won its official approval from the European Commission.
The drug is approved to treat enlarged spleens and related symptoms in adult myelofibrosis patients with moderate to severe anemia. Omjjara, also known as momelotinib, is the first drug specifically approved for this use in Europe, GSK said in a release.
"The availability of a single therapy for key manifestations of myelofibrosis is a clear step forward for eligible patients," Francesca Palandri, M.D., Ph.D., of the Italian hospital Policlinico Sant'Orsola-Malpighi, said in a statement.
- Meanwhile, GSK's label expansion bid for RSV vaccine Arexvy is moving ahead in Europe.
The European Medicines Agency has accepted the company's application for Arexvy as an RSV prophylactic for people 50 to 59, GSK said.
Last year, European officials approved the vaccine for the prevention of lower respiratory tract disease caused by RSV in people 60 and older.
GSK expects a decision on its new filing during the second half of this year.
UPDATED: Thursday, Jan. 25 at 1:30 p.m. ET
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Heron Therapeutics scored a label expansion for its extended-release local anesthetic, Zynrelef, (bupivacaine and meloxicam) to include soft tissue and orthopedic surgical procedures. The new FDA approval covers foot and ankle procedures, plus other operations in which direct exposure to articular cartilage is avoided, the company said in a press release.
According to Heron, Zynrelef is the only therapy for post-surgery pain management that’s demonstrated superiority to the current standard of care, bupivacaine, in late-stage trials.
With an expanded approval in hand, Heron expects Zynrelef will be eligible for use in an estimated 13 million U.S. procedures per year, an increase of 86% over prior indicated procedures. To nab its new approval, Heron ran tests studying the med's use in cesarean section, spinal surgery, augmentation mammoplasty and total shoulder arthroplasty procedures.
Zynrelef won its initial FDA approval in May 2021.
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Meanwhile, North Carolina’s Liquidia Corporation will have to wait a while longer to clinch an approval for its Yutrepia inhalation powder in pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD). Thursday, the FDA told the company it was “confirming the process for adding the PH-ILD indication as an amendment" to Yutrepia’s drug application, meaning the agency will miss its previously stated action date of Jan. 24.
Back in late 2021, the FDA granted Yutrepia a tentative approval to treat PAH to improve exercise ability in adult patients with certain symptoms. Seeking to add PH-ILD to Yutrepia’s label, Liquidia filed an amendment to its drug application in July 2023.
The FDA previously said Yutrepia could potentially add PH-ILD to its label without additional clinical trials.
UPDATED: Tuesday, Jan. 23 at 9:45 a.m.
- The FDA has missed its Jan. 22 target decision date for Theratechnologies' new formulation of lipodystrophy drug Egrifta (tesamorelin).
Theratechnologies said it has shown that its F8 formulation of tesamorelin is bioequivalent to the original F1 formulation, which is no longer available in the U.S. The company currently offers Egrifta SV, or an F4 formulation, which itself has shown bioequivalence to F1.
Compared with the F4 formulation, F8 tesamorelin is two times more concentrated, therefore requiring a smaller volume of administration. The new version comes in a multi-dose vial that can be reconstituted once per week.
The company said it will provide further information on the filing "in due course."
- In India, AstraZeneca has received approval from the Central Drugs Standard Control Organisation (CDSCO) to import and market andexanet alfa, Financial Express reports.
The drug, marketed in the U.S. as Andexxa, treats life-threatening or uncontrolled bleeding in patients who have received a Factor Xa inhibitor.
UPDATED: Thursday, Jan. 18 at 9:55 a.m.
- Roche's Tecentriq SC, the subcutaneous version of the company's blockbuster PD-L1 cancer therapy, has secured its first approval in Europe.
The European Commission approved the drug to treat certain patients with urothelial carcinoma, lung cancer, breast cancer and hepatocellular carcinoma. With the nod, Tecentriq SC becomes the first subcutaneous PD-1 or PD-L1 cancer medicine available in Europe, Roche said in a release.
More than 38,000 patients received the original, intravenous version of the drug in Europe last year. The IV approach takes 30 to 60 minutes, while the under-the-skin subcutaneous injection takes around five minutes, according to Roche.
Roche notes that the subcutaneous route of administration "offers the potential for a faster, more convenient alternative" to an intravenous infusion. The subcutaneous approach is "preferred by cancer patients, nurses and physicians," according to the Swiss drugmaker.
- In the United Kingdom, Santhera's Agramree has picked up approval to treat patients 4 and older with Duchenne muscular dystrophy.
The U.K.'s Medicines and Healthcare products Regulatory Agency adopted the view of the European Medicines Agency and recognized the drug's "clinically important safety benefits" over standard of care corticosteroids, plus its "similar efficacy," the company said in a release.
The nod follows approvals for the drug in Europe and the U.S.
European launches are progressing this quarter, Santhera said in the release, with Germany expected to be out ahead of other countries.
UPDATED: Thursday, Jan. 11 at 10:00 a.m.
- China's National Medical Products Administration has approved GSK's Nucala as an add-on maintenance treatment for patients with severe eosinophilic asthma who are 12 years of age and older.
With the nod, Nucala becomes the first targeted IL-5 biologic approved in China in this indication, GSK said in a release.
The approval is based on a China-only, 52-week phase 3 trial. The study found that patients who received GSK's drug experienced 65% fewer clinically significant exacerbations than those who received placebo.
In 2021, GSK's drug won approval in China to treat eosinophilic granulomatosis with polyangiitis.
- Mirati Therapeutics' KRAS-targeted therapy Krazati has won an approval in Europe to treat adults with KRASG12C-mutated advanced non-small cell lung cancer who've experienced disease progression after at least one prior systemic treatment.
In a registration-enabling phase 2 study, the drug met its endpoints of objective response rate and duration of response, the biotech said in its release.
The European Commission's conditional approval decision follows a positive recommendation from the Committee for Medicinal Products for Human Use in November 2023.
Meanwhile, Mirati is in the process of being bought out by Bristol Myers Squibb.
- Also in Europe, Stada and Alvotech have won regulatory clearance for the first biosimilar of Johnson & Johnson's Stelara.
Following a patent expiration in July, the approval "paves way for biosimilar competition in the approximately €2.5 billion" Stelara market in Europe, according to the partners.
In the U.S., Johnson's blockbuster immunology drug is expected to face competition early next year.
UPDATED: Tuesday, Jan. 9 at 2:45 p.m.
- Pfizer's PARP inhibitor Talzenna has gained European approval when used in combination with Pfizer and Astellas' Xtandi to treat adult patients with metastatic castration-resistant prostate cancer (mCRPC) in whom chemotherapy is not clinically indicated.
This approval makes Talzenna the first PARP inhibitor in Europe to be licensed in combination with Xtandi in mCRPC, Pfizer said in a release.
The European Commission approved Pfizer's application based on data from the phase 3 TALAPRO-2 trial, which showed that the combo cut the risk of disease progression or death in patients with mCRPC compared with placebo and Xtandi.
Last June, the FDA approved the combination to treat adults with HRR gene-mutated mCRPC.
- In another regulatory win for Pfizer, the FDA is reviewing the company's full approval application on Genmab-partnered Tivdak.
Under its priority review timeline, the FDA is assessing whether to convert Tivdak's accelerated approval into a full nod to treat patients with recurrent or metastatic cervical cancer who've had disease progression on or after first-line therapy.
The FDA plans to act on the filing by May 9, Pfizer said.
- Eisai and Biogen's Alzheimer's disease drug Leqembi has picked up an approval in China to treat patients with mild disease.
Eisai estimates there will be 17 million patients in China in 2024 who could be eligible for Leqembi based on the med's current label. And the med's popularity is expected to grow as the population ages.
"Eisai will focus on AD awareness via omnichannel systems and collaborate with specialists to improve the diagnostic environment," the company said.
- Amid Sarepta's early launch of Duchenne muscular dystrophy gene therapy Elevidys, the company is aiming for a significant label expansion.
In an update (PDF) at the J.P. Morgan Healthcare Conference, Sarepta argued that late-stage clinical data on the medicine "exceeded the threshold for substantial evidence of effectiveness" and that the drug's risk/benefit profile "remains favorable." Sarepta is expecting a decision by the FDA on its expansion bid by August, the company said.
Elevidys won an accelerated FDA approval back in June to treat ambulatory DMD patients ages 4 to 5 years with a confirmed mutation in the DMD gene. After that accelerated approval and subsequent trial results, the company is pursuing an FDA nod to treat all patients.
This week, analysts with Mizuho said they "believe the odds are high for the label expansion," according to Investing.com.
UPDATED: Friday, Jan. 5 at 9:35 a.m.
- After last year's approval from the U.S. FDA, Pfizer's RSV vaccine Abrysvo has secured an approval from Health Canada.
Regulators in Canada endorsed the vaccine for use in adults 60 years of age and older to prevent lower respiratory tract disease caused by RSV. In addition, the agency signed off on the vaccine's use in people who are between 32 and 36 weeks pregnant to protect their infants after they're born.
Canada's National Advisory Committee on Immunization is conducting its own review of the vaccine and will issue guidelines around using the vaccine in Canada.
GSK's Arexvy is also approved in Canada for use in people 60 and older.
- Ultragenyx's Evkeeza has picked up a coverage recommendation in England.
The National Institute for Health and Care Excellence (NICE) issued final draft guidance recommending the medicine, plus diet and other LDL-C therapies, for the treatment of adults and children 12 and older with homozygous familial hypercholesterolemia (HoFH).
Ultragenyx points out that the drug is the first angiopoietin-like 3 inhibitor for the disease.
In 2022, the company inked a deal with the drug's developer, Regeneron, to commercialize Evkeeza outside the U.S.
UPDATED: Wednesday, Jan. 3 at 9:30 a.m.
- After two decades of stagnation, the United Kingdom's Medicines & Healthcare products Regulatory Agency (MHRA) has welcomed a new endocrine therapy into the cancer fighting fold.
Now dubbed Korserdu, Menarini Group's small molecule drug has been cleared in the U.K. as the first medicine to treat ER+, HER2- advanced or metastatic breast cancer tumors with ESR1 mutations. The drug is specifically authorized for postmenopausal women and men who've already tried at least one line of endocrine therapy, including a CDK 4/6 inhibitor.
ESR1 mutations are acquired mutations that develop from exposure to endocrine therapy, which drive resistance to standard endocrine therapy.
Korserdu helped patients with ESR1 mutations hit a median progression-free survival of 3.8 months versus 1.9 months for those on standard of care. The treatment also reduced the risk of progression or death by 45% compared to standard of care.
Korserdu's U.K. green light comes several months after the medicine won marketing authorization in the European Union under the brand name Orserdu.
- Elsewhere, the European Medicines Agency (EMA) accepted Bristol Myers Squibb's application for potential approval of its next-generation tyrosine kinase inhibitor (TKI) repotrectinib. The drug is angling for approval in locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC) and NTRK-positive solid tumors.
BMS girded its application with data from the phase 1/2 Trident-1 trial, as well as the pediatric Care study. The clutch of clinical trials shows repotrectinib charted clinically meaningful response rates in patients across ROS1-positive NSCLC and NTRK-positive solid tumor cohorts.
Back in November, the U.S. FDA approved repotrectinib as Augtyro to treat adult patients with locally advanced or metastatic ROS1-positive NSCLC.
- Speaking of the FDA, the U.S. drug regulator late last month approved Coherus BioSciences' Udenyca Onbody delivery system for its biosimilar to Amgen's febrile neutropenia drug Neulasta.
Coherus' copycat, dubbed Udenyca, is given to patients the day after chemotherapy to curb infections caused by febrile neutropenia.
The company's Onbody delivery system tees up administration of pegfilgrastim in just five minutes. The delivery system also features an indicator and status light, plus an audio signal that helps patients confirm the dose has been administered.
Coherus expects to make its Udenyca Onbody delivery system available in the U.S. during 2024's first quarter.