Welcome to Fierce Pharma's regulatory tracker for the first half of 2025. On this page, we're recording the regulatory progress of in-market products, including expansions into key geographies and new indications. Be sure to come back regularly for the latest updates.
UPDATED: Monday, April 28 at 10:50 a.m. ET
Several new medicines scored positive recommendations at the recent meeting of Europe's Committee for Medicinal Products for Human Use (CHMP), as did eight biosimilars to Amgen’s popular bone meds Prolia and Xgeva.
Amgen’s Tepezza, a key component of the company's $27.8 billion buyout of Horizon Therapeutics, is on its way to becoming Europe’s first available treatment for thyroid eye disease after winning the CHMP’s backing. Tepezza is the world’s only approved drug for the disease, which can cause vision loss and facial disfigurement in severe cases.A new treatment for cystic fibrosis is also on the horizon in Europe, as Vertex Pharmaceuticals’ once-daily Alyftrek scored a CHMP endorsement after a phase 3 study found it to be as effective as Vertex’s established triple-combo Kaftrio/Trikafta. Alyftrek is another triple combo from Vertex that's intended for patients who have at least one non-class 1 mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
Jazz Pharmaceuticals' human epidermal growth factor receptor 2 (HER2)-targeted bispecific antibody, Ziihera, also received a positive CHMP opinion as a treatment for unresectable locally advanced or metastatic HER2-positive biliary tract cancer (BTC) that’s been previously treated with at least one prior line of systemic therapy. After the drug's final signoff at the European Commission, it's slated to become Europe’s first HER2-targeted therapy for the tough-to-treat cancer.
Last week's CHMP meeting also saw the committee endorse a number of new indications for approved drugs, including Argenx’s Vyvgart as a monotherapy for certain patients with chronic inflammatory demyelinating polyneuropathy (CIDP) and AstraZeneca’s Calquence in a new combination to treat previously untreated chronic lymphocytic leukaemia (CLL).
Eli Lilly’s Alzheimer’s disease treatment Kisunla, meanwhile, will get a second shot at a commercial future in Europe with a CHMP re-examination. The committee previously turned down the drug during its March meeting, citing safety concerns tied to the known side effect of amyloid-related imaging abnormalities. Now, CHMP will issue a final recommendation after it re-evaluates its opinion.
- Elsewhere, BridgeBio Pharma’s Bayer-partnered Beyonttra won the favor of the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA). The agency issued a marketing authorization for the drug as a treatment for wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). The nod follows a November FDA approval, which cleared the drug under the brand name Attruby.
UPDATED: Tuesday, April 22 at 1:15 p.m. ET
The U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) has signed off on Pfizer’s Hympavzi (marstacimab), a weekly injection which reduces bleeding episodes for patients with hemophilia A or B. .
It has been cleared for those age 12 and older who weigh at least 35 kilograms (77 pounds) and have not developed antibodies to previous inhibitor treatments. Provided in an auto-injector, the drug is designed to be a convenient alternative to periodic infusions of proteins that boost blood clotting.
In the fourth quarter of last year, the FDA and European Commission endorsed the treatment, which is the first of its kind as it targets the Kunitz-2 domain of the tissue factor pathway inhibitor (TFPI).
In developing the treatment, Pfizer trailed Novo Nordisk’s investigational TFPI monoclonal antibody concizumab, but the FDA rejected Novo's prospect in May 2023 because of dosing and manufacturing issues. In December of last year, the U.S. regulator approved the treatment as Alhemo.
Also last year, Pfizer gained approval for hemophilia A drug Beqvez in the U.S. and in Europe, where it is known as Durveqtix.
UPDATED: Friday, April 18 at 10:15 a.m. ETAkeso is broadening its horizons with a nod for its IL-12/IL-23 monoclonal antibody psoriasis drug in China, marking the company’s first approved drug in the autoimmune space.
China’s National Medical Products Administration cleared the drug, ebdarokimab, to be used in patients with moderate-to-severe plaque psoriasis after five clinical studies proved its short-term and long-term efficacy, Akeso said in a press release.
By week 16, 79.4% patients who received 135 mg doses of ebdarokimab saw at least a 75% improvement in their Psoriasis Area and Severity Index (PASI 75) scores from baseline. In addition, 77.9% of those who received maintenance therapy through week 52 achieved the PASI 75 benchmark. Significant improvements in quality of life were also observed, Akeso pointed out.
Ebdarokimab only needs to be dosed four times a year, making it a convenient and accessible option, according to Akeso. Elsewhere in its non-oncology portfolio, the company received a nod in China for its hypercholesterolemia med, ebronucimab, in September.
The FDA tweaked the label for Bristol Myers Squibb’s heart med Camzyos to simplify the required twice-yearly echo monitoring and remove the previous contraindications for CYP2C19 inhibitors and CYP3A4 inhibitors, allowing a broader group of patients to use the med.
Patients who once needed echo monitoring every 12 weeks now only need it every 6 months, while those who are on moderate CYP2C19 inhibitors and strong CYP3A4 inhibitors are now eligible to be prescribed Camzyos.
The drug is a key product in BMS' growth portfolio and is the first and only cardiac myosin inhibitor indicated to treat those with symptomatic New York Heart Association (NYHA) class II-III obstructive hypertrophic cardiomyopathy in the U.S. Camzyos carries a boxed warning for the risk of heart failure.
The CDC’s Advisory Committee on Immunization Practices (ACIP) updated its recommendations for Valneva’s chikungunya vaccine, Ixchiq, adding a precaution for those ages 65 and older and including a consideration for those travelling to a country without an outbreak.
The committee kept its preexisting recommendation for those travelling to a country where there is a chikungunya outbreak, but noted that the single-dose vaccine “may be considered” for those planning extended travel to a country or territory without an outbreak.
That group of U.S. travelers is considered at an elevated risk if they plan to stay for six months or more, ACIP said.
As for users 65 and older, the precaution was tacked on due to an ongoing CDC investigation of six cases of serious adverse events, including five hospitalizations, that occurred in people ages 67 to 86 years after vaccination. No such events have been reported since January 2025. The events were flagged in the Vaccine Adverse Event Reporting System, which is an early warning system that can’t determine if adverse events were caused by a vaccine. All of the hospitalized individuals had pre-existing comorbidities and ACIP concluded that no casual association with the vaccine could be found.
UPDATED: Wednesday, April 16 at 8:35 a.m. ET
Biocon Biologics, seeking to launch a biosimilar version of Regeneron's blockbuster eye disease medicine Eylea, has secured a launch date for its product.
Biocon Biologics expects to launch its biosimilar, Yesafili, "in the second half of calendar year 2026 or earlier in certain circumstances," the company said in a Wednesday press release.
The launch date comes courtesy of a settlement and licensing agreement that the company inked with Regeneron.
Previously, Amgen launched the first U.S. biosimilar of Regeneron's Eylea.
Merck, known as MSD outside the U.S., has secured a new approval for its Gardasil 9 vaccine in China.
Regulators in the country approved the shot's use in males between the ages of 16 and 26, according to China Daily. The product is now the first nine-valent HPV shot to be endorsed in males and females in China, the publication notes.
Earlier this year, Merck's four-valent HPV shot, Gardasil, secured approval to be used in males in China.
As Valneva works to grow the market for its chikungunya vaccine Ixchiq, the company has secured its first marketing authorization in an endemic nation.
Brazil's health regulatory agency, ANVISA, endorsed the shot for use in people 18 and older, Valneva said in a Monday press release.
Besides Brazil, regulators in the U.S., Europe, Canada and the United Kingdom have approved the vaccine.
UPDATED: Monday, April 14 at 10:20 a.m. ET
With a green light from the European Commission (EC), Roche’s Columvi (glofitamab) has earned the distinction of becoming Europe’s first bispecific antibody for diffuse large B-cell lymphoma (DLBCL) in patients who haven’t responded to initial treatment.
The drug was specifically approved in tandem with the chemotherapy cocktail of gemcitabine and oxaliplatin (GemOx) to treat adults with relapsed or refractory DLBCL not otherwise specified who aren’t good candidates for autologous stem cell transplant (ASCT).
The new clearance comes after Columvi initially won conditional marketing authorization in Europe for relapsed or refractory DLBCL patients after two or more lines of systemic therapy in July 2023.
The EC based its earlier-line approval on results from Roche’s late-stage Starglo study, in which Columvi plus GemOx yielded a statistically significant overall survival improvement over rituximab and GemOx.
Digging deeper into the numbers, the Roche combo helped reduce patients’ risk of death by 41% compared to the control and also met secondary endpoints tied to measures like progression-free survival.
In follow-up analyses at around 20.7 months, patients who’d received the Columvi cocktail lived for a median 25.5 months, nearly double the 12.9 months seen in the rituximab-GemOx group.
DLBCL is an aggressive form of lymphoma that makes up one of the most prevalent types of blood cancer among adults. There are around 38,000 people diagnosed with the disease in Europe each year and approximately four out of 10 DLBCL patients will relapse after first-line treatment, Roche estimates.
Elsewhere in Europe, the EC has kicked off its “final process” in deciding on the label of Eisai and Biogen’s Alzheimer’s disease drug Leqembi (lecanemab) in the bloc.
Leqembi initially won a thumbs-up from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) back in November, albeit in a restricted patient population. The EC then tasked the CHMP with considering new safety data on the drug that surfaced after the initial recommendation.
By late February, the CHMP had signed off on Leqembi once again, punting the final approval decision back to the commission.
“Following the EC deliberations on the [marketing authorization application (MAA)] in its Standing Committee in March 2025, the Appeal Committee has conducted its deliberations and did not reach a decision,” Eisai explained in a press release Monday.
Over in the U.K., patients may soon have access to a new targeted breast cancer treatment under England’s National Health Service (NHS), thanks to a recommendation from the region’s cost watchdog, the National Institute for Health and Care Excellence (NICE), endorsing AstraZeneca’s Truqap (capivasertib) as part of a combination regimen for an advanced form of the disease.
The recommendation, which pairs the AKT inhibitor Truqap with AstraZeneca’s estrogen receptor antagonist Faslodex (fulvestrant), specifically covers use of the cocktail in patients with HR-positive, HER2-negative breast cancer that has progressed after earlier endocrine treatment.
Clinical trials have suggested the combination could give patients an additional four months on average before their conditions progress compared to Faslodex alone.
“While it won’t be suitable for everyone, giving patients more time before more intensive therapies are required is an important part of our drive to personalize cancer care and improve quality of life for patients wherever possible,” Peter Johnson, national clinical director for cancer at NHS England, said of the recommendation in a statement.
In the U.S., South Korea’s Celltrion has received a thumbs-up from the FDA granting interchangeability status for its biosimilar Yuflyma, which references AbbVie’s immunology powerhouse Humira, multiple Korean news outlets reported Monday.
Interchangeability, which is a highly coveted designation within the biosimilar world, allows pharmacists to swap out biosimilars for their branded counterparts right at the counter, without the need for doctor sign-off. The classification makes it about as simple to dispense interchangeable biosimilars as it is to provide patients with generic small-molecule drugs.
Yuflyma launched in the U.S. in the summer of 2023 and currently operates across eight of Humira’s established indications.
To win the interchangeability approval, Celltrion furnished the FDA with data from a Humira-Yuflyma switching study in 367 patients with moderate to severe plaque psoriasis, Korea Biomedical Review reported early this week. The trial found that switching between the drugs did not affect their safety, efficacy or immunogenicity, the local news outlet explained.
UPDATED: Thursday, April 10 at 8:56 a.m. ET
Regulators in China have approved the country's first gene therapy. The product, called dalnacogene ponparvovec (BBM-H901), has been endorsed by the National Medical Products Administration to treat adult patients with moderate to severe hemophilia B. The one-time therapy is developed and manufactured by local biotech Belief BioMed and will be commercialized by Takeda.
Similar to CSL's FDA-approved Hemgenix, BBM-H901 uses an adeno-associated virus vector to deliver a functional human factor IX gene. Patients with hemophilia B have insufficient levels of the gene, leaving them susceptible to severe bleeding.
Among 26 Chinese patients enrolled in a single-arm phase 3 trial, BBM-H901 helped lower the mean annualized bleeding rate (ABR) to 0.6 after a year of follow-up. This included 21 (80.8%) patients who had no bleeding events at all. The ABR was significantly lower than that observed in externally identified comparable patients who were on prophylactic treatment.
Mean factor IX expression at week 52 reached 55.08 IU/dL, which is considered a normal level. And no patients suffered serious adverse events.
Traditional factor replacement therapies require frequent intravenous infusions, and patients' factor levels fluctuate with this approach, Zhang Lei, from the Blood Disease Hospital of the Chinese Academy of Medical Sciences, said in a statement Thursday.
Further, some patients are at risk of developing inhibitors against factor replacement treatment, Lei noted. Lei is the lead investigator of BBM-H901's clinical program.
"The approval of BBM-H901 has brought revolutionary hope to Chinese patients with hemophilia B," Lei continued. "By a single dosing, the risk of bleeding and joint damage can be reduced, and patients are back to a normal life."
"The approval of BBM-H901 will further enrich our portfolio in hemophilia and rare disease areas, and will help more patients to benefit from innovative treatments," Takeda's China president, Sean Shan, said in a statement.
The price of BBM-H901 is expected to be released at a later time. In the U.S., CSL's Hemgenix comes with a list price of $3.5 million per treatment.
About seven years after an initial accelerated approval, the FDA has granted Bayer's Vitrakvi a full approval for certain patients whose solid tumors have an NTRK gene fusion. The approval covers patients who have no satisfactory alternative treatments or who have progressed on previous therapy.
The full nod puts Vitrakvi one step ahead of Roche's Rozlytrek, which scored its own accelerated approval for NTRK solid tumors in 2019. Rozlytrek, but not Vitrakvi, is also cleared for ROS1-positive non-small cell lung cancer.
Vitrakvi's traditional approval is based on data from three single-arm trials, coded Loxo-TRK-14001, Scout and Navigate, comprising a total of 339 patients. Pooled efficacy results showed an objective response rate of 60%, including a 24% complete response rate.
- Biocon Biologics has won FDA approval for Jobevne, a biosimilar to Roche's Avastin. The product marks he company's seventh biosimilar approved in the U.S.
UPDATED: Tuesday, April 8 at 2:57 p.m. ET
Bristol Myers Squibb’s oncology combo of Opdivo (nivolumab) plus Yervoy (ipilimumab) scored another FDA approval Tuesday, this time as a first-line treatment for adults and kids ages 12 and older with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) colorectal cancer.
At the same time, the FDA converted the accelerated approval for Opdivo monotherapy in MSI-H or dMMR metastatic colorectal cancer into a standard approval. Opdivo is approved in that indication for patients whose disease has progressed after treatment with fluoropyrimidine, oxaliplatin, and irinotecan.
The FDA gave the thumbs up to Bristol’s oncology cocktail after reviewing data from the phase 3 CHECKMATE-8HW trial, which tested Opdivo and Yervoy against both chemotherapy and Opdivo by itself.
In the study, Opvido plus Yervoy cut the risk of disease progression or death by 38% compared to Opdivo monotherapy in patients who hadn’t tried an immunotherapy before. Compared to chemotherapy, Opdivo plus Yervoy slashed the risk of disease progression or death by 79% in the first line setting.
Since 2015, BMS’ Opdivo-Yervoy combo has racked up an impressive number of approvals across melanoma, renal cell carcinoma, colorectal cancer, lung cancer and mesothelioma. The latest nod—which came two months ahead of schedule—marks the ninth for Opdivo-based regimens in gastrointestinal cancers, the company said.
Vertex has picked up a label expansion for its cystic fibrosis drug Kaftrio in combination with ivacaftor in the European Union (EU), where the regimen is now approved in all patients ages 2 and older with at least one non-class 1 mutation in the cystic fibrosis conductance regulator (CFTR) gene.
Patients in places like Austria, Denmark, Ireland, Norway, Sweden and Germany should be able to access Kaftrio “shortly” under the expanded indication, thanks to existing reimbursement deals and access provisions in those countries, Vertex explained in a press release.
“We have been working for years to bring treatment options to all people with cystic fibrosis, including those with ultra rare mutations,” Carmen Bozic, M.D., Vertex’s chief medical officer, said in a statement, adding that the approval ensures that even more cystic fibrosis patients can now benefit from Vertex’s medicine.
Also in the EU, the European Commission has granted approval to AbbVie’s JAK inhibitor Rinvoq for the treatment of adults with giant cell arteritis (GCA). The green light makes Rinvoq the first oral JAK inhibitor cleared in the EU—plus Iceland, Liechtenstein and Norway—for the autoimmune disease.
GCA, which tends to affect patients older than 50 years of age, results in inflammation of the temporal and other cranial arteries, as well as the aorta and other large and medium arteries, AbbVie said in a release.
The condition can cause headaches, jaw pain and muscle aches and potentially lead to vision loss.
The European Commission based its approval on results from AbbVie’s phase 3 SELECT-GCA trial, in which once-daily Rinvoq and a 26-week steroid taper regimen bested placebo on metrics of sustained remission, disease flare reduction, cumulative steroid exposure and more.
UPDATED: Monday, April 7 at 12:30 p.m. ET
Johnson & Johnson’s subcutaneous version of Darzalex can now treat more European patients with newly diagnosed multiple myeloma.
Specifically, EU regulators signed off on the drug as part of a quadruplet regimen that includes Takeda’s Velcade, Bristol Myers Squibb’s Revlimid and the corticosteroid dexamethasone (daratumumab-VRd).
The treatment regimen won approval (PDF) from the European Commission following a previous nod for patients who are eligible for autologous stem-cell transplant (ASCT), meaning it’s now the only anti-CD38 antibody indicated to treat all patients in the frontline setting regardless of transplant eligibility.
In the phase 3 CEPHEUS study, daratumumab-VRd met its primary endpoints, with 60.9% of patients taking the quadruplet regimen meeting an overall minimal residual disease-negativity rate at a median follow-up of 59 months. For patients on the triplet combo of Velcade, Revlimid and dexamethasone, the number was 39.4%.
The company is also seeking approval of the regimen in the U.S. and filed a bid with the FDA in September for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for ASCT or for whom ASCT is deferred. In Europe, the latest nod marks the drug’s ninth indication, five of which are in the frontline setting.
With the FDA acceptance of Teva's label expansion filing, the company could be on its way to making its migraine med Ajovy the first calcitonin gene-related peptide (CGRP) antagonist approved to prevent episodic migraines in children and adolescents.
The company seeks to expand Ajovy’s label to include episodic migraine prevention in patients ages 6 to 17 years old who weigh 99 pounds or more. A wider label for the drug could be a “much-needed solution” for the underserved population, global R&D head and chief medical officer Eric Hughes, M.D., Ph.D., noted in a company release.
In Teva’s phase 3 SPACE trial, subcutaneous Ajovy showed statistically significant improvements in reducing monthly migraine days and monthly headaches over placebo during a 12-week period. Episodic migraines in children can lead to school absences and missed social events, causing impaired educational performance and disrupted social lives.
The nod would add a big patient pool to the drug’s previous 2018 approval for migraine prevention in adults. Ajovy works by targeting the underlying cause of migraines and is the only anti-CGRP treatment available for the condition that’s offered in both quarterly and monthly dosing options.
Johnson & Johnson also won a European Commission approval for the subcutaneous version of its Rybrevant combined with its Lazcluze as a first-line treatment for certain adults with advanced non-small cell lung cancer (NSCLC), matching the already approved indications of the intravenous formulation.
The drug combo can treat NSCLC patients with epidermal growth factor receptor (EGFR) exon 19 deletions (ex19del) or exon 21 L858R substitution mutations, and Rybrevant can be used on its own in those with advanced NSCLC with activating EGFR exon 20 insertion mutations after platinum-based therapy fails.
In studies, the under-the-skin formulation was non-inferior to the established intravenous formulation and made for an overall response rate of 30% compared with IV’s 33%. Administration time for the subcutaneous route was just five minutes and results proved a five-fold reduction in infusion-related reactions over the other version.
UPDATED: Friday, April 4 at 9:55 a.m. ET
AstraZeneca and Daiichi Sankyo's Enhertu has scored approval in Europe as a monotherapy to treat certain adults with HR-positive, HER2-low or HER2-ultralow breast cancer.
Specifically, the approval covers patients who have received at least one endocrine therapy in the metastatic setting and who are not considered suitable for endocrine therapy as the next line of treatment.
The approval, based on results from the DESTINY-Breast06 trial, makes Enhertu the first HER2-directed therapy for this group of patients, AstraZeneca said in a press release. In the trial, Enhertu demonstrated a 38% reduction in the risk of disease progression or death versus chemotherapy.
“Enhertu continues to open up new approaches to the diagnosis and treatment of patients with metastatic breast cancer," AZ's oncology and hematology business unit head, Dave Fredrickson, said in a statement. "This approval underscores the importance of testing metastatic breast cancer tumours for any IHC staining to identify patients with HR-positive, HER2-low or HER2-ultralow disease who may be eligible for Enhertu once sustained responses are no longer achieved with endocrine-based therapy.”
Also in Europe, AstraZeneca's Imfinzi has scored a new approval as part of a perioperative regimen in certain patients with lung cancer.
The new nod allows Imfinzi to be used in combination with chemotherapy to treat adults with resectable non-small cell lung cancer (NSCLC) at high risk of recurrence and no epidermal growth factor receptor (EGFR) mutations or anaplastic lymphoma kinase (ALK) rearrangements.
For this regimen, patients receive Imfinzi and neoadjuvant chemo before surgery and Imfinzi alone as a monotherapy after surgery.
Regulators reviewed results from the AEGEAN trial for this approval. At a planned interim analysis, the study showed that the Imfinzi regimen demonstrated a 32% reduction in the risk of disease recurrence, progression events or death versus neoadjuvant chemotherapy alone.
UPDATED: Tuesday, April 1 at 4:25 p.m. ET
Apellis’s Empaveli is up for a speedy FDA review in rare kidney diseases C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN).
The agency granted the company’s application its priority review designation and assigned a decision date of July 28. Empaveli is currently approved to treat aroxysmal nocturnal hemoglobinuria (PNH) and could reach another 5,000 U.S. patients if it tacks on C3G and IC-MPGN uses to its label. About half of patients living with the diseases develop kidney failure within five to ten years of diagnosis, according to Apellis.
Empaveli proved its worth across three key markers of disease in the phase 3 VALIANT study, demonstrating proteinuria reductions, stabilization of kidney function, and substantial clearance of C3c staining. Apellis is looking forward to working with the FDA to “make this treatment available to patients in need as quickly as possible,” Cedric Francois, M.D., Ph.D., said in a press release.
In addition, the FDA communicated that it does not plan to hold an advisory committee meeting for the application, according to a note from Mizuho analyst Graig Suvannavejh, Ph.D. This development "bodes well for an ultimate approval," Suvannavejh wrote to clients.
The FDA approved a new defense against potential future mpox outbreaks with the freeze-dried formulation of Bavarian Nordic’s smallpox and mpox vaccine Jynneos.
The freeze-dried version of the vaccine offers advantages in transportation, storage conditions and shelf life that make it ideal for long-term stockpiling, while the previously approved liquid-frozen formulation comes with specific cold-chain requirements.
Jynneos was first approved in the U.S. in 2019, but Bavarian Nordic has been supplying its liquid-frozen vaccine to the U.S. government for stockpiling since 2010. The first freeze-dried doses should be delivered later this year, according to the company.
Pfizer’s respiratory syncytial virus (RSV) vaccine Abrysvo won a European label expansion that allows the vaccine’s use as a prophylactic against RSV-related lower respiratory tract disease (LRTD) in a larger population.
Compared with a prior adult label that covered people 60 and older, the European Commission has amended its authorization for Abrysvo to include people ages 18 to 59. The expansion allows the vaccine the broadest RSV vaccine indication in Europe and was based on Pfizer’s phase 3 MONeT study, which tested the vaccine in those at risk of RSV-associated LRTD due to certain chronic medical conditions.
Abrysvo can also be given to pregnant people who are between weeks 24 and week 36 gestation to help protect infants from LRTD caused by RSV at birth up until six months of age.
Eisai’s Alzheimer’s disease drug Leqembi is still in limbo in Europe, with an appeals committee now expected to make the final decision on approval after the European Commission passed the drug over.
The European Commission was meant to make the final call on the drug after the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recently reaffirmed its previously issued positive opinion, which came after a reevaluation. The commission's standing committee has since deliberated on the decision, but opted to refer the case to the appeal committee based on “the results” of those talks, Eisai’s European partner BioArtic reported.
CHMP in November endorsed Leqembi in a limited population of Alzheimer’s patients with mild cognitive impairment or dementia who carry only one or no copies of the ApoE4 gene, since those with two copies are at a greater risk of amyloid-related imaging abnormalities side effects.
UPDATED: Friday, March 21 at 10:30 a.m. ET
Hutchmed won a conditional approval for Epizyme’s lymphoma med Tazverik in China, marking the country’s first approved EZH2 inhibitor and the company’s first foray into hematological malignancies.
China’s National Medical Products Administration (NMPA) specifically signed off on the drug’s use in adult patients with relapsed or refractory (R/R) follicular lymphoma (FL) with EZH2 mutation who have received at least two prior systemic therapies. Under its agreement with Epizyme, Hutchmed is responsible for developing, manufacturing and commercializing the med in China Mainland, Hong Kong, Macau and Taiwan, while Epizyme remains the marketing authorization holder of Tazverik in China.
Epizyme, an Ipsen subsidiary through a 2022 merger that centered on Tazverik, launched the therapy in the U.S. after winning FDA accelerated approvals in 2020 covering patients with R/R FL and epithelioid sarcoma. Despite a slow launch, the oncology med quickly posed a rival to Gilead Sciences’ competing Zydelig. Eisai, meanwhile, commercializes the drug in Japan. Tazverik garnered $46.7 million euros ($50.5 million) for Ipsen in 2024.
Hutchmed looks to make Tazverik available to patients “as soon as possible,” chief medical officer Michael Shi, M.D., Ph.D., said in a statement. FL is the second most common subtype of non-Hodgkin’s lymphoma and is prevalent in China, with an estimated 81,000 new cases in China and 78,000 in the U.S. in 2022.
UPDATED: Wednesday, March 19 at 9:05 a.m. ET
The National Institute for Health and Care Excellence (NICE) has recommended Gideon Richter’s Ryeqo as the first daily pill for endometriosis. The treatment is a combination of the hormone medications relugolix, estradiol and norethisterone.
While endometriosis affects around 1.5 million women in the U.K., the drug may help about 1,000 women per year by NICE's estimate. The drug has been approved for routine use on the NHS in England and Wales, but it's only for patients who have failed other medical and surgical treatments.
The backing from the drug cost watchdog follows a previous rejection and comes after Gideon Richter provided new evidence on Ryeqo's effectiveness and value for money, NICE said.
"This new treatment marks a potential step-change in how we manage endometriosis, putting control back in patients' hands while ensuring value for the taxpayer," Helen Knight, NICE's director of medicines evaluation, said in a statement.
Biogen said its Skyclarys has won an approval in Canada to treat Friedreich's ataxia, a rare, progressive, debilitating and life-shortening neurodegenerative disease. An estimated 300 to 750 people live with the disease in Canada.
The drug is the first treatment available in the country to slow the progression of the disease, according to the company. In the pivotal MOXIe Part 2 trial, patients treated with Skyclarys showed a statistically significant improvement in modified Friedreich's Ataxia Rating Scale (mFARS) scores compared with those on placebo after 48 weeks.
UPDATED: Monday, March 17 at 10:00 a.m. ET
The FDA has accepted and granted priority review status to Bayer’s application seeking approval for Kerendia in heart failure patients with mildly reduced (HFmrEF) or preserved ejection fraction (HFpEF).
Ejection fraction measures the heart’s ability to pump oxygen-rich blood throughout body. A lower ejection fraction means that a patient’s heart is struggling to perform as intended. Of the estimated 6.7 million people living with heart failure in the U.S., roughly 55% have a left ventricular ejection fraction (LVEF) greater than or equal to 40%. That population would be covered by the label Bayer is seeking.
Bayer based its approval bid on data from the phase 3 FINEARTS-HF trial, which showed that Kerendia helped reduce patients’ risk of cardiovascular death and heart failure events by 16% versus placebo.
Kerendia is the first nonsteroidal mineralocorticoid receptor antagonist (MRA) to meet a primary composite cardiovascular endpoint in a late-stage study investigating heart failure patients with an LVEF of more than 40%, Bayer said when it originally disclosed its FINEARTS-HF data last summer.
A heart failure approval would be a major boon for Kerendia, which Bayer has predicted could eventually generate $3 billion in annual sales. If approved, the drug would likely compete with SGLT2 inhibitors already on the market for heart failure, such as Eli Lilly and Boehringer Ingelheim’s Jardiance and AstraZeneca and Bristol Myers Squibb’s Farxiga.
Following a thumbs up from Europe’s human medicines committee in early February, the European Commission has formally approved AstraZeneca’s PD-L1 inhibitor Imfinzi for the treatment of limited-stage small cell lung cancer (LS-SCLC) in adults whose disease hasn’t gotten worse after platinum-based chemoradiation therapy.
The European Commission granted its approval after reviewing data from AZ’s phase 3 ADRIATIC study, which showed that Imfinzi reduced patients’ risk of death by 27% versus placebo and delivered an estimated median overall survival (OS) time of 55.9 months compared to 33.4 months in the control group.
Further, an estimated 57% of Imfinzi patients were alive at three years, compared to 48% of patients who received placebo, AZ said. Imfinzi also curbed the risk of disease progression by 24%, the company added.
Imfinzi already boasts an LS-SCLC approval in the U.S. and several other countries. Currently, AZ is also working to secure additional green lights in places like Japan.
As for the latest regulatory nod, AZ estimates there are around 8,000 people treated for LS-SCLC “across the five major European countries each year.” The condition often recurs and progresses rapidly, even after patients have achieved an initial response to standard care chemotherapy and radiation, AZ noted.
Travere Therapeutics submitted an application to the FDA seeking priority review of its primary immunoglobulin A nephropathy (IgAN) drug Filspari in focal segmental glomerulosclerosis (FSGS).
FSGS is a rare proteinuric kidney disorder that affects around 40,000 patients—both kids and adults—in the U.S. The disorder is marked by the progressive scarring of the kidney and often leads to kidney failure, Travere said in a press announcement.
Aside from other common symptoms like swelling and hypertension, FSGS is characterized by proteinuria, where protein leaks into the urine due to a breakdown in the kidney’s filtration mechanism. Once in the urine, protein is considered toxic to other parts of the kidney and is believed to fuel further disease progression, Travere explained.
Travere is padding its application with data from the phase 3 DUPLEX and phase 2 DUET studies of Filspari in FSGS, which have shown that the drug triggered “rapid, superior and sustained reductions in proteinuria when compared maximum labeled dose irbesartan” in kids and adults with the rare kidney disorder, the company said.
The FDA has also informed the company that it not longer needs to implement a risk evaluation and mitigation strategy (REMS) concerning the potential danger of embryo-fetal toxicity in patients taking Filspari.
UPDATED: Friday, March 14 at 9:25 a.m. ET
China's National Medical Products Administration (NMPA) has approved Innovent's Sycume as a treatment for thyroid eye disease (TED).
The drug, also known as teprotumumab N01, becomes the first anti-insulin-like growth factor 1 receptor (IGF-1R) antibody to score approval in the country.
In a press release, Innovent said the drug's approval "has ended a 70-year drought of no new treatment option for TED in China."
In the U.S., teprotumumab is marketed by Amgen as Tepezza.
In Europe, officials have expanded the label for Bristol Myers Squibb's cell therapy Breyanzi.
Specifically, the European Commission approved the drug for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy.
The approval is based on results from the global phase 2 study called TRANSCEND FL, which enrolled patients with relapsed or refractory indolent non-Hodgkin lymphoma (NHL), including FL. In the third-line-plus treatment setting, 97.1% of patients responded to the drug, and 94.2% of patients achieved a complete response.
Before this approval, Breyanzi had scored EU nods to treat certain patients with diffuse large B-cell lymphoma, high grade B-cell lymphoma and primary mediastinal large B-cell lymphoma.
UPDATED: Thursday, March 13 at 8:30 a.m. ET
Kelun-Biotech's TROP 2 ADC sacituzumab tirumotecan (sac-TMT) has scored its second approval in China.
The country's National Medical Products Administration (NMPA) approved the drug to treat certain adults with EGFR-positive locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC). The nod covers the drug's use in patients who have progressed after treatment with EGFR-tyrosine kinase inhibitor (TKI) therapy and platinum-based chemotherapy, Kelun said in a press release.
With the approval, sac-TMT becomes the first TROP2 ADC approved to treat lung cancer anywhere in the world, according to the company.
In a pivotal study, the drug "demonstrated a statistically significant and clinically meaningful improvement" in objective response rate, progression-free survival and overall survival compared with docetaxel, Kelun said. The study tested the drugs in patients with locally advanced or metastatic EGFR-mutant NSCLC who failed after prior treatment with an EGFR-TKI and platinum-based chemo.
The approval is sac-TMT's second in the country after the NMPA approved it to treat certain patients with triple negative breast cancer last year.
In 2022, Merck & Co. licensed ex-China rights to the drug.
UPDATED: Friday, March 7 at 9:35 a.m. ET
The European Commission (EC) has signed off on Bristol Myers Squibb’s immunotherapy combination of Opdivo and Yervoy to treat first-line patients with advanced or unresectable hepatocellular carcinoma (HCC).
The nod was based on Opdivo and Yervoy showing an overall survival edge in the CheckMate-9DW trial over an investigator's choice of oral treatments.
The Opdivo-Yervoy combo also faces an FDA decision date of April 21 to treat unresectable HCC. In 2020, the FDA granted accelerated approval to Opdivo-Yervoy to treat second-line advanced HCC.
Opdivo and Yervoy were originally approved to treat melanoma, in 2014 and 2011 respectively. In combination, they have been blessed by the FDA in four other cancer types: renal cell carcinoma (2018), colorectal cancer (2018), mesothelioma (2020) and lung cancer (2020).
Approximately 90% of liver cancer diagnoses are of the HCC type, BMS said. Liver cancer is the world’s third leading cause of cancer deaths.
- The FDA has expanded its approval for ALK’s immunotherapy Odactra for house dust mite (HDM) allergies to children ages 5 through 11. The under-the-tongue tablet was originally approved for adults in 2017 and then for adolescents ages 12 to 17 in 2023.
UPDATED: Thursday, March 6 at 9:45 a.m. ET
ARS Pharmaceuticals needle-free epinephrine option, neffy nasal spray, can now be used to treat type 1 allergic reactions including anaphylaxis in children.
To win the pediatric nod from the FDA, ARS ran studies proving that pharmacokinetic and pharmacodynamic responses from neffy in both children and adults were consistent with that of standard epinephrine injection products. The label expansion comes after the drug’s August approval as the first major innovation in epinephrine delivery in more than 35 years.
Needle-free delivery is especially significant in the pediatric population as many children and caregivers fear needle-based auto-injectors, standing to delay lifesaving treatment.
Human factor studies also show that children as young as 10 can use the nasal spray effectively and that untrained individuals, such as babysitters or teachers, can also correctly administer the drug. In the U.S., nearly 4 out of every 10 epinephrine prescriptions are for children under the age of 18 and some one in 13 children have severe food allergies, with more than 40% having experienced severe reactions, according to ARS.
UPDATED: Wednesday, March 5 at 10:10 a.m. ET
Roche's supplemental biologics application for Gazyva to treat lupus nephritis has been accepted for review by the FDA, the company said in a Wednesday press release.
The filing leverages results from the phase 3 REGENCY study, which showed that 46.4% of patients with lupus nephritis who received Gazyva plus standard care achieved a complete renal response after 76 weeks. For patients who received standard care alone, the number was 33.1%, according to study results published last month.
First approved in 2013, Gazyva is already cleared to treat chronic lymphocytic leukemia and follicular lymphoma in certain situations.
The FDA is expected to make a decision on the lupus nephritis filing by October, according to Roche.
UPDATED: Monday, March 3 at 4:30 p.m. ET
Despite a reconsideration after a prior rejection, Australian drug regulators ultimately passed on Eisai's BioArtic-partnered Alzheimer’s disease treatment Leqembi.
The Therapeutic Goods Administration (TGA) of Australia first decided against approval for patients with early Alzheimer’s disease in October. Eisai then requested reconsideration of the decision in December, proposing the same narrow indication given to the drug by the Medicines and Healthcare products Regulatory Agency (MHRA) and European Medicines Agency (EMA). Those approvals stipulate that Leqembi can only be used in patients with one or no copies of the ApoE4 gene due to safety concerns linking two copies of the gene to increased risks of brain swelling and bleeding, a side effect known as Amyloid Related Imaging Abnormalities (ARIAs).
However, the TGA did not agree with the safety profile of Leqembi in ApoE4 heterozygotes, or those with one copy of the ApoE4 gene. Instead, the regulator offered an alternative narrow indication covering only ApoE4 noncarriers. Eisai floated other indications instead, including one that would keep the ApoE4 noncarrier and heterozygote patient populations but require heterozygotes to receive treatment in specialist centers with expert physician supervision. Still, the TGA rejected those options.
BioArtic, Eisai’s long-time research partner that originally developed the antibody that became Leqembi, said it’s “very disappointed” by TGA’s decision, CEO Gunilla Osswald said in a statement.
Eisai said it remains committed to ensuring eligible Australians can access the treatment and is “exploring options” to achieve this including a potential review by Australia’s Administrative Review Tribunal.
UPDATED: Friday, Feb. 28 at 10:30 a.m. ET
The CDC said it's investigating five hospitalizations related to heart and nervous system events following vaccination with Valneva's Ixchiq among people 65 years and older.
Ixchiq became the first FDA-approved chikungunya vaccine in November 2023. The CDC currently recommends the live-attenuated shot to be used for adults traveling to a country or territory where there is a chikungunya outbreak.
Based on information from the CDC, at least four of the five individuals had also received other vaccines, and most of them had significant underlying medical conditions, a Valneva spokesperson said in a statement to Fierce Pharma.
"Valneva has not identified any safety signal concerns through post-marketing safety monitoring, including periodic safety reports and routine signal detection activities, and Ixchiq’s safety profile remains consistent with clinical trial data, unchanged and positive," the spokesperson said.
The CDC said findings from its investigation will be discussed with experts at an Advisory Committee on Immunization Practices (ACIP) meeting. This year's first ACIP meeting, which was supposed to take place this week, was postponed with no new time provided by the CDC.
The U.K. has approved Moderna's respiratory syncytial virus (RSV) vaccine mRESVIA to protect patients ages 60 and over against lower respiratory tract disease caused by the virus.
In a study conducted in about 37,000 older adults, people who received mRESVIA had a 79% reduction in their risk of getting lower respiratory tract disease caused by RSV, compared with those who received placebo, around four months after vaccination.
The shot will be manufactured at the Moderna Innovation and Technology Centre in Oxfordshire, which will be fully operational later this year, CEO Stéphane Bancel said in a statement.
As is the case in the U.S., Moderna's mRNA shot will compete with GSK's Arexvy and Pfizer's Abrysvo in the U.K. as well.
UPDATED: Thursday, Feb. 27 at 8:32 a.m. ET
After being turned away by the FDA nearly one year ago, Regeneron this week said that the U.S. agency has accepted for review its biologics license application for bispecific antibody odronextamab.
The FDA is expected to act on the application by July 30, Regeneron said in a Wednesday press release.
In March of 2024, the agency rejected an accelerated approval filing for the drug based on the timeline of Regeneron's ongoing confirmatory trials. That decision spurred some confusion at Regeneron—and beyond—about a new requirement from the agency that confirmatory trials be "well underway" by the time it awards accelerated approvals.
Early this year, the FDA sought to clear up the issue with new guidance on its expectations around accelerated approvals and confirmatory trial timelines.
Nevertheless, Regeneron's review for the drug to treat patients with relapsed/refractory (R/R) follicular lymphoma (FL) after two or more lines of systemic therapy now appears back on track. In its latest press release, Regeneron said the resubmission "follows the achievement of an FDA-mandated enrollment target for the Phase 3 confirmatory trial." As the company notes, the enrollment target was the "sole approvability issue identified by the FDA" in its last submission.
In Europe, regulators last year approved the drug for patients with R/R FL and R/R diffuse large B-cell lymphoma after two or more lines of systemic therapy. The medicine carries the brand name Ordspono in the region.
In a phase 2 study, 80% of patients with FL who were treated with odronextamab responded to the therapy, with 74% achieving a complete response.
UPDATED: Tuesday, Feb. 25 at 9:30 a.m. ET
Merck's Keytruda, already boasting some 40 FDA approvals, has picked up a priority review in a proposed new use.
The company's filing for the drug as part of a perioperative regimen for patients with resectable locally advanced head and neck squamous cell carcinoma has been accepted for a speedy review, Merck said on Tuesday.
The filing seeks approval for Keytruda as a neoadjuvant treatment prior to surgery, followed by its administration as an adjuvant treatment in combination with standard of care radiotherapy—with or without cisplatin—after surgery. Afterward, the company's filing proposes Keytruda being used as a single agent.
Merck's application leverages results from the KEYNOTE-689 trial, which showed that the perioperative regimen significantly reduced the risk of disease recurrence, worsening or death in certain patients with resected HNSCC compared with traditional postsurgical radiotherapy alone.
The FDA is expected to act on the application by June 23.
In Japan, Sanofi's Sarclisa has picked up an approval to treat newly diagnosed patients with multiple myeloma.
Japan's Ministry of Health, Labour and Welfare (MHLW) approved the drug as part of a combination with bortezomib, lenalidomide, and dexamethasone (VRd) in the patient group based on results from the phase 3 IMROZ trial.
The study showed that Sarclisa's addition to VRd significantly improved progression-free survival compared with VRd alone in patients with newly diagnosed, transplant-ineligible multiple myeloma.
Sarclisa launched in 2020 in Japan and is approved as a part of four different regimens in the country, Sanofi noted in a press release.
UPDATED: Monday, Feb. 24 at 10:52 a.m. ET
Gilead's application for twice-yearly lenacapavir has been accepted by the European Medicines Agency for accelerated review as an HIV pre-exposure prophylaxis (PrEP) option. Simultaneously, the drug will be assessed under EU-Medicines for all, which can be used to facilitate expedited review processes in countries outside the EU, including in low- and lower-middle-income countries.
The FDA has put lenacapavir's PrEP application under priority review last week with a target decision date set for June 19, 2025.
Several companies have launched their biosimilar products referencing Johnson & Johnson's star inflammatory disease drug Stelara in the U.S., following Amgen's January launch of Wezlana as the first biosimilar referencing the star IL-12/23 inhibitor.
Teva and Alvotech just rolled out Selarsdi covering all indications and presentations of the original Stelara. Teva can start to claim interchangeability after an exclusivity period for Wezlana ends on April 30.
Sandoz announced the launch of its Samsung Bioepis-partnered Pyzchiva, which the Swiss company views as a key driver in its global growth strategy. Sandoz touts Pyzchiva's extended stability, including the ability to be re-refrigerate, as an advantage over the original Stelara.
In addition, Biocon Biologics has also entered the U.S. Stelara biosimilar market with Yesintek. It marks the Biocon subsidiary's "first product launch in the United States since becoming a fully integrated global biosimilars organization," CEO Shreehas Tambe said in a release.
The FDA has granted priority review to Bristol Myers Squibb's application for Opdivo and Yervoy in first-line unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) colorectal cancer (mCRC). The goal date is June 23, 2025.
The filing was based on data from the phase 3 CheckMate-8HW trial. The combo lowered the risk of disease progression or death by 38% versus Opdivo alone in MSI-H/dMMR mCRC, according to an update of the study presented in January.
The FDA has updated the label of Indivior's Sublocade for severe opioid use disorder.
The new label allows healthcare providers to start treatment with Sublocade after a single dose of transmucosal buprenorphine and a one-hour observation period. This rapid initiation "may lessen some of the practical obstacles to treatment induction," according to Indivior.
In addition, the FDA has approved additional injection sites for Sublocade, an extended-release formulation of buprenorphine. The drug can now be administered in the abdomen, thigh, buttock or back of the upper arm.
UPDATED: Friday, Feb. 21 at 9:30 a.m. ET
Gilead's seladelpar has secured a conditional marketing nod in Europe to treat adults with primary biliary cholangitis (PBC).
The approval covers seladelpar's use in combination with ursodeoxycholic acid (UDCA) for those who have an inadequate response to UDCA alone, or as a monotherapy for people who can't tolerate UDCA.
In the U.S., seladelpar picked up FDA approval last year and is branded as Livdelzi.
A chronic autoimmune disease, PCB affects roughly 22 out of 100,000 people in Europe. The disease is more common in women and causes liver damage that can progress to liver failure if untreated. Symptoms include chronic itch and fatigue, and there is no cure for the disease.
The approval is largely based on results from the phase 3 Response study, which showed that roughly three times as many patients on seladelpar achieved a composite biochemical response than those on placebo. The conditional approval will be contingent on positive results from future confirmatory studies.
In Japan, authorities have approved CSL's subcutaneous Andembry for the prevention of acute attacks of hereditary angioedema (HAE).
The drug offers patients the first pre-filled pen presentation for monthly subcutaneous administration for long-term prophylaxis of HAE, CSL said in a press release.
The Japanese nod adds to the drug's other approvals in Europe, Australia and the U.K.
UPDATED: Thursday, Feb. 20 at 10:00 a.m. ET
Bristol Myers Squibb's Breyanzi has won reimbursement backing from England's National Institute for Health and Care Excellence (NICE) in second-line large B-cell lymphoma, becoming the first CAR-T therapy to be recommended by the agency for this cancer type.
The endorsement comes after NICE had originally rejected Breyanzi in October 2024 during its draft assessment. The drug cost watchdog changed its mind after BMS "offered an improved commercial arrangement" to the NHS based on its 297,000 pound sterling (about $375,000) list price per person, according to a press release.
About 600 people in England could benefit from the therapy per year, according to NICE, which called the one-time CAR-T treatment "a significant advance in lymphoma care."
In another reversal, NICE has also changed a previous rejection and signed off on UCB's Fintepla as an add-on treatment for seizures associated with Lennox-Gastaut syndrome (LGS) in people ages 2 years and above. The drug already has coverage under NHS for treating seizures from Dravet syndrome.
LGS is a rare but severely debilitating form of epilepsy that begins in early childhood. By NICE's estimate, about 1,400 patients live with the disease in its jurisdiction.
NICE changed its mind after UCB offered "an improved discount" to the drug's list price, the agency noted. Using a cost comparison approach, UCB convinced NICE's drug reviewers that Fintepla provides similar or greater health benefits compared to cannabidiol plus clobazam—which is already covered for LGS in the NHS—at a similar or lower cost.
The drug is covered only if the frequency of drop seizures is checked every six months, at which point the frequency is reduced by at least 30% compared with before starting treatment.
UPDATED: Tuesday, Feb. 18 at 11:04 a.m. ET
Merck’s Welireg snagged its first two approvals in Europe, with the nods covering certain patients with von Hippel-Lidau (VHL) disease and advanced clear renal cell carcinoma (RCC).
The European Commission specifically approved the medicine as a treatment for those with VHL disease who have associated, localized RCC, central nervous system hemangioblastomas or pancreatic neuroendocrine tumors. The drug is approved in cases when localized procedures are unsuitable, making Welireg the first available treatment for VHL-associated tumors in Europe.
The med also won a nod for adult patients with advanced clear cell RCC that has progressed following two or more lines of therapy, including a PD-1/L1 and at least two VEGF-targeted therapies.
The European nods, which are conditional authorizations, follow similar approvals in the U.S. Merck will make the drug commercially available individual European countries after the complication of national reimbursement procedures, among other launch timing factors, the company said.
Gilead’s twice-yearly pre-exposure prophylaxis (PrEP) candidate lenacapavir is on the FDA’s review table with a decision date of June 19.
The drug, which is currently approved as Sunlenca for multi-drug resistant HIV, would be the first twice-yearly PrEP option on the market. The company’s FDA bid is the first step of its detailed global launch and access strategy.
Gilead’s lenacapivir application is backed by two phase 3 trials, one of which showed an impressive 100% risk reduction. The company describes the med as a “foundation” for potential future HIV therapies, it said in a release.
Sanofi and Regeneron’s immunology powerhouse Dupixent is aiming to secure a new use in chronic skin disease bullous pemphigoid (BP) with an FDA decision expected by June 20.
If approved, the med would be the only targeted BP treatment available in the U.S. The disease is hallmarked by intense itch and blisters and impacts a population of around 27,000 adults, typically elderly adults, whose condition is not controlled by systemic corticosteroids.
The companies’ FDA bid has been granted priority review and is based on a study of 106 adults with moderate-to-severe BP. In the trial, five times more patients who received Dupixent achieved sustained disease remission compared to those on placebo, according to Sanofi.
UPDATED: Friday, Feb. 14 at 9:20 a.m. ET
CSL's hereditary angioedema (HAE) therapy Andembry has secured approval from the European Commission.
The drug is the first once-monthly treatment targeting factor XIIa to prevent HAE attacks in patients 12 and older.
In a phase 3 trial, the drug significantly reduced investigator-confirmed HAE attacks per month versus placebo, according to results published in The Lancet in April 2023.
Last month, regulators in Australia and the U.K. signed off on the medicine.
"Andembry, CSL's first approved recombinant monoclonal antibody discovered and developed entirely by CSL, underscores our more than 40-year legacy in HAE research and treatment optimization and our decades-long journey to bring this innovation to patients," Bill Mezzanotte, M.D., CSL's head of R&D, said in a press release.
Now, CSL plans to engage in access and reimbursement negotiations with national authorities across Europe as the company progresses with the rollout.
Also in Europe, officials signed off on CSL and Arcturus Therapeutics' self-amplifying mRNA COVID-19 vaccine, Kostaive, for use in individuals ages 18 and older.
The approval is based on results from an integrated phase 1/2/3 study and late-stage booster trials, the partners said in a press release. Late last year, the European Medicines Agency recommended the vaccine for approval.
Besides Europe, the vaccine is approved in Japan.
Galderma's efforts to expand the market for Nemluvio are bearing more fruit.
In Europe, officials approved the subcutaneous drug for the treatment of moderate-to-severe atopic dermatitis in patients ages 12 and older. In addition, the drug scored an endorsement to treat prurigo nodularis in adults.
In the late-stage Arcadia and Olympia clinical trial programs, the drug "significantly improved itch, skin lesions and sleep disturbance, in patients with moderate-to-severe atopic dermatitis and adults with prurigo nodularis, respectively," Galderma said in a Friday press release.
The drug is also approved in the U.S. and is under review in Canada, Brazil, South Korea and other countries.
China has approved its 22nd PD-1/L1 product. The approval went to SinoCellTech's finotonlimab for its use alongside chemo in first-line head and neck cancer.
In a China phase 3 trial, the combo reduced the risk of death by 27% versus chemo alone, extending the median overall survival by 3.6 months to 14.1 months, according to results published in Nature Medicine.
The go-ahead marks the first for a PD-1/L1 inhibitor that covers all head and neck cancer patients in China. In the country, Merck & Co.'s Keytruda is allowed as a monotherapy only in PD-L1-positive disease, SinoCellTech noted in a securities filing (Chinese).
UPDATED: Wednesday, Feb. 12 at 10:20 a.m. ET
Acoramidis, the transthyretin amyloid cardiomyopathy (ATTR-CM) drug developed by BridgeBio and licensed by Bayer in Europe, has been approved by the European Commission. Bayer plans to make the transthyretin stabilizer available in Europe under the brand name Beyonttra in the first half of this year.
The approval is based on results from the phase 3 ATTRibute-CM study trial showing that Beyonttra significantly reduced the composite endpoint of all-cause mortality and cardiovascular-related hospitalizations compared with placebo. During the open-label extension phase of the study, Beyonttra demonstrated a relative risk reduction in deaths of 34% after 42 months.
Beyonttra's launch is one of Bayer's “must-win battles” as the German company navigates the loss of exclusivity of its top-selling drug, blood thinner Xarelto, Stefan Oelrich, head of Bayer’s pharma division, said at the J.P. Morgan Healthcare Conference in January.
Travere Therapeutics said it has aligned with the FDA on its plan to submit Filspari for traditional approval in focal segmental glomerulosclerosis (FSGS), a rare kidney disease.
The move comes after a Filspari phase 3 trial in that indication failed on its primary endpoint of eGFR slope, a measurement of kidney disease progression. It also comes as findings from a collaborative global project called ARASOL linked a decrease in proteinuria over 24 months to a reduction in the risk of kidney failure in FSGS. Filspari has shown a benefit in lowering proteinuria.
Travere plans to file this application around the end of March using existing data from the phase 3 DUPLEX and phase 2 DUET studies. The company will request a priority review, which Leerink Partners analysts believe is likely to be granted as there are no approved therapies for FSGS.
Given that the understanding around proteinuria as a potential full approval endpoint is new, Leerink analysts said it would not be surprising if the FDA wanted to discuss the results during an advisory committee meeting.
An approval in FSGS would be "transformational" for Filspari and Travere, Leerink said in a Tuesday note, because it could give the drug $1.1 billion in peak sales across the U.S. and EU based on a "relatively conservative peak penetration" of less than 20%. For Filspari sales, the FSGS indication could eventually surpass IgA nephropathy, for which Filspari is currently approved and is starting to face more competition, according to Leerink Partners.
UPDATED: Monday, Feb. 10 at 9:45 a.m. ET
Bristol Myers Squibb’s cancer combo Opdivo plus Yervoy has scored an approval recommendation in the E.U., thanks to an endorsement from the EMA’s Committee for Medicinal Products for Human Use (CHMP) as a first-line treatment in adults with unresectable or advanced hepatocellular carcinoma.
With the thumbs up from CHMP, the Opvido-Yervoy combo now moves forward to the European Commission (EC) for a final approval decision.
The CHMP based its green light on results from the combo’s phase 3 CheckMate -9DW trial, in which patients on Opdivo and Yervoy charted a median overall survival of 23.7 months, compared to 20.6 months in patients who received an alternative regimen of Lenvima (lenvatinib) or sorafenib.
BMS’ cancer combo is up for a similar approval in the U.S., with the FDA scheduled to make its decision on a potential first-line unresectable HCC nod by April 21.
The drugmaker also won a CHMP endorsement for its CAR-T therapy Breyanzi to treat adults with relapsed or refractory follicular lymphoma (FL) who’ve previously tried two or more systemic therapies.
BMS used data from its phase 2 TRANSCEND FL study to support the recommendation. In the trial, Breyanzi charted an overall response rate of 97.1% and a complete response rate of 94.2%, which was sufficient to meet the study’s primary and secondary endpoints, BMS said in a release.
Breyanzi already carries multiple oncology approvals in Europe, including in relapsed or refractory diffuse large B-cell lymphoma and high grade B-cell lymphoma.
Late last month, the FDA gave a thumbs up to German drugmaker medac’s alkylating agent Grafapex, also known as treosulfan, alongside the antimetabolite medicine fludarabine, as a preparative regimen for allogeneic hematopoietic stem cell transplantation (alloHSCT) in adults and kids ages one and older with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).
Alkylating agents cause breakage of DNA strand to help thwart the ability of cancer cells to multiply.
Medac’s approval was backed up by a study looking primarily at overall survival in 570 adults ages 18 to 70 years old with AML or MDS who were randomized to receive either Grafapex or busulfan with fludarabine as a preparative regimen for allogeneic transplant.
UPDATED: Wednesday, Feb. 5 at 9:05 a.m. ET
Novartis' Kisqali has secured a label expansion from the U.K.'s Medicines and Healthcare products Regulatory Agency (MHRA).
The new approval covers Kisqali's use in combination with an aromatase inhibitor (AI) in patients with HR+/HER2- early breast cancer at high risk of recurrence.
Previously, the drug was approved by the MHRA to treat advanced breast cancer, according to a Feb. 4 press release from Novartis.
The latest approval is based on results from the phase 3 NATALEE trial, which showed that the addition of Kisqali to treatment with an AI cut the risk of invasive disease, recurrence, or death compared to AI treatment alone in patients with HR+/HER2- early breast cancer.
Last fall, the drug picked up an FDA nod in patients with HR+/HER2- early breast cancer based on the NATALEE study results.
Vaccine specialist Valneva has picked up an approval from the MHRA, as well.
The agency approved the company's chikungunya vaccine, Ixchiq, for use in people 18 years of age and older. Late-stage trial data show the vaccine "induces a rapid and strong immune response," according to a press release from the company.
Valneva manufactures the shot at its facility in Livingston, Scotland.
Previously, the vaccine secured approvals in the United States, Canada and Europe.
UPDATED: Friday, Jan. 31 at 9:35 a.m. ET
Vertex's groundbreaking launch of Casgevy keeps racking up more milestones around the globe.
Friday, the company said it has reached a reimbursement agreement with NHS England that'll allow eligible patients with sickle cell disease access to the CRISPR/Cas9 gene-edited therapy. Along with the NHS England agreement, the National Institute for Health and Care Excellence (NICE) has signed off on the drug's use, Vertex said in a press release.
The reimbursement deal follows a similar agreement, unveiled last summer, for the drug in patients with transfusion-dependent beta thalassemia.
“We are pleased to have reached this new agreement that ensures both eligible SCD and TDT patients can now be treated with Casgevy, recognizing the value a one-time treatment can provide to patients, their families and the healthcare system," Vertex International senior vice president Ludovic Fenaux said in a statement.
In its approval bid for Fabhalta in C3 glomerulopathy, Novartis will no longer face an FDA advisory committee meeting, CEO Vas Narasimhan said on Friday.
The advisory committee meeting had been scheduled for next month but will no longer take place.
Speaking with members of the media on Friday, Narasimhan said the "FDA no longer deemed it necessary to hold an advisory committee meeting."
"We think it's based on, you know, the overall data package we've been able to generate, and the additional follow on data that we've provided the agency," the CEO continued. So we look forward to now bringing forward that medicine as a third indication approval for Fabhalta."
Amid Europe's ongoing regulatory review of Eisai and Biogen's Alzheimer's disease medicine Leqembi, the European Commission (EC) has flagged new safety information for review.
In November, Europe's Committee for Medicinal Products for Human Use (CHMP) recommended market approval of the drug. Since then, EC has asked the committee to "consider information on the safety of lecanemab that became available after the adoption of the CHMP opinion," the partners said in a Friday press release.
The companies didn't reveal the nature of the new safety information flagged by the EC. CHMP will dig into the new info during a meeting next month, according to the release.
"We believe that the EC’s requests can be addressed with existing information and will be evaluated by the CHMP because of the clear and sufficient information available," the partners said in the release. "We will continue to work closely with the authorities toward approval in the EU."
UPDATED: Monday, Jan. 27 at 10:43 a.m. ET
Merck’s Welireg is up for a potential FDA label expansion by May 26. The agency granted a priority review to the company's bid to expand the drug's label into advanced, unresectable, or metastatic pheochromocytoma and paraganglioma (PPGL), which would add to currently approved indications in advanced renal cell carcinoma and von Hippel-Lindau disease.
Welireg generated $139 million in sales during 2024’s third quarter and is the only approved HIF-2α inhibitor in the U.S.
Merck’s application in PPGL is based on objective response rate and duration of response rate data from the phase 2 LITESPARK-015 trial, which enrolled 322 patients and will be presented at an upcoming medical meeting, according to the company.
Up to 2,000 new cases of the tumors are diagnosed yearly in the U.S.
Merck is evaluating Welireg across other rare oncologic diseases both on its own and in combination with other therapies.
The FDA is evaluating Alvotech and Teva’s proposed biosimilar to Johnson & Johnson’s Simponi and Simponi Aria.
Alvotech, which is responsible for development and manufacturing under its longstanding collaboration with Teva, announced positive top line results from its confirmatory trial of the Simponi copycat back in April. The partners expect the FDA to complete its review process in the fourth quarter of 2025.
Simponi was first approved in 2009 and treats a variety of inflammatory conditions including rheumatoid arthritis, psoriatic arthritis and ankylosing spondylitis, among others. Simponi is an injected drug, while Simponi Aria is the infused version. The franchise picked up $2.19 billion for J&J in 2023 sales.
Alvotech and Teva's product is the first biosimilar to the blockbuster arthritis med to be accepted for review.
UPDATED: Thursday, Jan. 23 at 10:00 a.m. ET
Biogen said its applications for a high-dose version of the spinal muscular atrophy med Spinraza have been accepted for review by the FDA and the European Medicines Agency. The new regimen comprises a more rapid loading regimen of two 50 mg doses given two weeks apart and a maintenance regimen given at 28 mg every four months.
Currently, the drug's FDA-approved loading regimen features three doses given 14 days apart, plus a fourth dose 30 days after the third. The drug is currently approved in 12 mg doses.
The high-dose applications are based on results from the phase 2/3 Devote trial, which favored the high-dose version versus an external sham control and the currently available dose on the measure of patients' reduction in plasma neurofilament light chain (NfL), a marker for neurodegeneration.
The higher dose regimen also reduced the risk of death or permanent ventilation by 68% relative to sham and 30% compared with the 12 mg regimen.
The high-dose Spinraza is Biogen's answer to competitors such as Novartis' gene therapy Zolgensma.
Amneal has won FDA approvals for generics referencing AbbVie's Alzheimer's combo drug Namzaric and Novartis' cancer therapy Afinitor Disperz. Amneal has launched the Namzaric generic with 180-day market exclusivity.
Separately, Amneal said it has won tentative approval from the FDA for a generic to Bausch Health's star irritable bowel syndrome med Xifaxan. Bausch Health has previously reached settlement agreements with Teva, Sun Pharma and Sandoz, pushing their generic entry dates into 2028 or upon an earlier launch of a rival generic product.
Xifaxan is one of 15 drugs up for the second cycle of Medicare price negotiations under the Inflation Reduction Act.
UPDATED: Tuesday, Jan. 21 at 8:57 a.m. ET
The European Commission has approved Johnson & Johnson's combination of Lazcluze and Rybrevant for the first-line treatment of certain adults with advanced non-small cell lung cancer (NSCLC), the company said in a press release.
Specifically, the combo is approved in Europe for patients with epidermal growth factor receptor (EGFR) exon 19 deletions (ex19del) or exon 21 L858R (L858R) substitution mutations.
In approving the use, EU regulators reviewed results from the phase 3 MARIPOSA study. The trial showed that the combination reduced the risk of disease progression or death by 30% compared with AstraZeneca's Tagrisso in patients with newly diagnosed, EGFR-mutant non-small cell lung cancer. The results yielded a U.S. approval for the J&J combination last summer.
Earlier this month, J&J touted an overall survival win from the study. At the time, a company executive heralded the regimen as "a new standard of care for patients with EGFR non-small cell lung cancer."
Also in Europe, officials have endorsed GSK's Jemperli to treat all adult patients with primary advanced or recurrent endometrial cancer, including those with mismatch repair proficient (MMRp)/microsatellite stable (MSS) tumors.
MMRp and MSS cases represent approximately 75% of patients diagnosed with endometrial cancer, GSK said in a press release.
The approval covers Jemperli's use in combination with chemotherapy.
Late last year, Europe's Committee for Medicinal Products for Human Use (CHMP) recommended the label expansion.
In England, the National Institute for Health and Care Excellence (NICE) recommended AstraZeneca's Tagrisso for treating people with EGFR mutation-positive non-small-cell lung cancer (NSCLC) after surgery.
The recommendation means that hundreds of patients will benefit from routine access to the drug, which is taken daily alongside chemotherapy, according to a notice from the Roy Castle Lung Cancer Foundation.
Previously, EGFR mutation-positive NSCLC patients relied on chemotherapy to prevent cancer recurrence after surgery, according to the foundation.
“I am pleased we have been able to recommend that this targeted treatment for a specific gene mutation of lung cancer will be routinely available on the NHS," NICE's director of medicines evaluation, Helen Knight, said in a statement.
UPDATED: Thursday, Jan. 16 at 10:25 a.m. ET
Eli Lilly's Omvoh has picked up its second major FDA nod.
After the IL-23 inhibitor's approval in 2023 to treat ulcerative colitis, the agency has now signed off on Omvoh to treat Crohn's disease. Specifically, the FDA approved Omvoh to treat moderately to severely active Crohn's disease in adults.In a phase 3 study, 53% of patients treated with Omvoh achieved clinical remission after a year of treatment. That number compared with 36% for those on placebo.
In addition, 46% of patients treated with Omvoh had visible healing of the intestinal lining after a year. In the placebo group, the number was 23%.
The trial enrolled patients with moderately to severely active Crohn's disease who had experienced an inadequate response, loss of response or who could not tolerate corticosteroids, immunomodulators and/or biologics.
With the FDA nod, Lilly's drug will go up against some heavy hitters in the Crohn's space, including Johnson & Johnson's Stelara and AbbVie's duo of Skyrizi and Rinvoq.
UPDATED: Wednesday, Jan. 15 at 8:12 a.m. ET
Sanofi's Sarclisa has gained approval from China's National Medical Products Administration (NMPA) to treat adults with multiple myeloma who've received at least one prior line of therapy, including lenalidomide and a proteasome inhibitor.
The approval covers Sarclisa's use in combination with pomalidomide and dexamethasone (Pd).
Regulators in China endorsed the drug based on results from the phase 3 ICARIA-MM study, as well as bridging data from a China-based study, Sanofi said in a press release. In ICARIA-MM, the addition of Sarclisa to Pd resulted in a 40% reduction in patients' risk of disease progression or death.
"We look forward to continuing to build strong partnerships with the medical community, local companies, and authorities in China as we work to bring more innovative treatments to patients," Sanofi's head of oncology, Olivier Nataf, said in a statement.
As Biogen and Eisai work to grow Leqembi in Alzheimer's disease, the FDA has accepted a biologics license application for the drug's subcutaneous maintenance dosing option.
With a potential approval, Leqembi would become the first anti-amyloid therapy to offer at-home maintenance dosing.
The FDA is set to make a decision on the filing by August 31, according to a Biogen press release.
UPDATED: Thursday, Jan. 9 at 11:15 a.m. ET
England’s National Institute for Health and Care Excellence (NICE) recommended AstraZeneca’s Lynparza for use in the region’s National Health Service, clearing the way for around 1,200 eligible patients to reap the benefits of the treatment.
The recommendation covers Lynparza’s use in patients with HER2-negative, locally advanced or metastatic breast cancer who have BRCA1 or BRCA2 mutations and have had chemotherapy. NICE based its decision in part on results from the company’s OlympiAD phase 3 study, which proved that the drug could cut the risk of disease worsening or death by 42%.
NICE had originally begun its assessment of the treatment in that indication in 2018, but ended it citing a lack of evidence submitted by the company. This time around, NICE assessed the med using a faster cost comparison process that compared Lynparza to Pfizer’s Talzenna in benefits and cost.
Stallergenes Greer’s peanut allergy treatment Palforzia scored a label expansion in Europe, extending the drug’s use to toddlers aged 1 through 3 years old.
Palforzia is now cleared for use in those aged 1 to 17 years old and is the only approved oral immunotherapy in the U.S. and Europe for toddlers with a confirmed peanut allergy diagnosis. The drug is meant to gradually increase the body’s ability to tolerate small amounts of peanuts through carefully controlled initial dose escalation and up-dosing.
The European Commission based its nod on the company’s POSEIDON phase 3 study, which was a double-blind, placebo-controlled food challenge after six months of treatment.
Agios Pharmaceuticals’ Pyrukynd is up for an FDA decision in non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia on Sept. 7.
The company filed its bid for approval last month after finding success in two phase 3 trials assessing the drug in the non-transfusion-dependent and transfusion-dependent populations. Thalassemia is a rare, inherited blood disorder with limited treatment options. The disease is hallmarked by the abnormal production of hemoglobin, which causes chronic anemia and can lead to other complications.
Pyruknd won its first approval in 2022 for pyruvate kinase (PK) deficiency, marking the first drug approved to treat the disease.
Merck’s Gardasil HPV vaccine won approval from the National Medical Products Administration of China for males aged 9 to 26 years old, making the first option of its kind available for the country's male population.
The vaccine was previously approved for use in females who are 9 to 45 years of age and has helped protect over 50 million women in China from certain HPV-related cancers and diseases since its first approval, Merck’s president of Human Health International, Joseph Romanelli, said in a release.
Gardasil has recently seen slumping sales in China, where it's being marketed by Merck’s regional commercial partner Zhifei Biological Products. Rob David previously called the expansion to the male population a “significant opportunity.”
UPDATED: Tuesday, Jan. 7 at 3:57 p.m. ET
The FDA tacked on additional warning information to the prescribing information for Agios’ pyruvate kinase (PK) deficiency drug Pyrukynd, flagging instances of liver deficiency in certain situations.
The label update follows liver injury observations in patients who received Pyrukynd at a dose higher than is recommended for PK deficiency, according to an SEC filing from the company.
The drug was approved by the FDA in 2022 as the first medicine to treat hemolytic anemia in adults with PK deficiency. Besides this use, the company is exploring expansions into beta thalassemia and sickle cell disease.
The updated "Warnings and Precautions" section urges prescribers to run liver tests on patients before initiating treatment and monthly for the first six months of treatment. Patients should stop treatment if hepatic injury is suspected.
Bayer is vying for a third indication for its Nubeqa (darolutamide) in China, this time for its use in combination with androgen deprivation therapy (ADT) in patients with metastatic hormone-sensitive prostate cancer.
The company filed an application for the label expansion with China’s National Medical Products Administration, leveraging data from its phase 3 ARANOTE trial. In the study, Nubeqa and ADT significantly reduced the risk of radiological progression or death by 46% compared with placebo plus ADT.
The nod would add to the drug’s two existing approvals in China, which cover other prostate cancer patient populations. It is estimated that the prevalence of prostate cancer in China will exceed 161,000 cases annually by 2026, according to Bayer.
Dizal’s non-small cell lung cancer (NSCLC) candidate sunvozertinib will get a speedy FDA review after the agency granted priority review to the company’s new drug application.
The company is specifically going after an approval to treat locally advanced or metastatic non-small cell lung cancer (NSCLC) in patients with epidermal growth factor receptor (EGFR) exon 20 insertion mutations whose disease has progressed on or after platinum-based chemotherapy.
Sunvozertinib won its world-first approval in China in 2023.
UPDATED: Monday, Jan. 6 at 10:25 a.m. ET
Amgen's DLL3-targeted bispecific antibody Imdylltra, known as Imdelltra in the U.S., has won a conditional marketing authorization from the U.K.'s Medicines and Healthcare products Regulatory Agency as a third-line therapy for small cell lung cancer.
The conditional nod is based on results from the phase 2 DeLLphi-301 trial, which showed that the T-cell engager triggered a tumor response rate of 41%, with the median response lasting 9.7 months. It follows an FDA approval in May 2024.
China has approved Astellas' first-in-class Claudin 18.2 drug Vyloy, following similar moves by drug regulators in Japan and the U.S.
The drug's Chinese indication is in first-line, CLDN18.2-positive, HER2-negative advanced gastric or gastroesophageal junction adenocarcinoma. The CLDN18.2 biomarker is expressed by about 35% of Chinese stomach cancer patients, according to Astellas.
The approval was backed by two global phase 3 trials, dubbed Glow and Spotlight, which evaluated Vyloy in combination with the chemotherapy regimens CAPOX and mFOLFOX6, respectively. Both trials linked the drug's chemo combo to longer life expectancy and progression-free survival time compared with chemo alone.
UPDATED: Friday, Jan. 3 at 2:45 p.m. ET
GSK’s IL-5 inhibitor Nucala scored a nod in China to treat people with inadequately controlled chronic rhinosinusitis with nasal polyps (CRSwNP), a condition that impacts nearly 30 million people in China.
The drug was approved by China’s National Medical Products Administration for use as an add-on therapy along with intranasal corticosteroids for patients with CRSwNP who don’t achieve adequate disease control with systemic corticosteroids or surgery.
The indication is Nucala’s third in China and comes after prior approvals in eosinophilic asthma and eosinophilic granulomatosis with polyangiitis.
An FDA advisory committee panel is scheduled to discuss the merits of Novartis’ oral factor B inhibitor Fabhalta in the rare kidney disease C3 glomerulopathy (C3G).
The Cardiovascular and Renal Drugs Advisory Committee will meet on February 24, according to an FDA document (PDF). The drug was approved by the agency in 2023 as a treatment for paroxysmal nocturnal hemoglobinuria, winning the title of the first oral monotherapy approved for the rare blood disorder. Fabhalta received a second nod for immunoglobulin A nephropathy (IgAN) in August.
In studies, Fabhalta proved that it could reduce proteinuria for C3G patients, a marker for delaying progression to kidney failure. So far, there are no approved treatments for the ultra-rare disease, which causes about 50% of patients to progress to kidney failure within ten years of diagnosis.
UPDATED: Thursday, Jan. 2 at 1:45 p.m. ET
- Shortly before the new year, Merck & Co. scored an approval for its potential blockbuster Winrevair in the U.K. The pulmonary arterial hypertension (PAH) drug, which also goes by the name sotatercept, was originally picked up via Merck’s $11.5 billion acquisition of Acceleron back in 2021.
The U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) approved Winrevair in combination with other PAH therapies to improve exercise capacity in adult patients who live with moderate or marked limitations of physical activity.
Patients with PAH suffer from high blood pressure in the blood vessels that supply the lungs, which can ultimately damage the right side of the heart. Winrevair, for its part, targets the causes of PAH responsible for the narrowing of the arteries in the lungs, in turn making it easier for the heart to pump blood to the pair of organs, according to the U.K. government’s release.
The MHRA based its approval on a study of 323 PAH patients that saw Winrevair best placebo at improving exercise ability. The main measure of effectiveness in the trial was the difference in distance patients could walk in six minutes before and after treatment.
At the trial’s 24-week treatment mark, patients on Winrevair plus other PAH medicines improved their walking distance by around 34 meters, versus one meter in patients who received placebo.
Merck’s U.K. nod for Winrevair follows green lights from the U.S. FDA last March and the European Commission in August.
At the time of the drug’s U.S. approval last year, J.P. Morgan’s Chris Schott estimated Winrevair could eventually reel in peak sales between $3 billion and $4 billion.