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Last year, Novo Nordisk's new hemophilia treatment Concizumab won approval from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) after being rejected by the US Food and Drug Administration (FDA). Meanwhile, PTC Therapeutics' treatment for Duchenne Muscular Dystrophy, which performed poorly in a recent FDA review, is in trouble. Novo Nordisk's anti-tissue factor pathway Inhibitor (TFPI) antibody has been recommended by the CHMP for marketing as A drug to prevent bleeding in hemophilia A or B patients using FVIII or FIX inhibitors. The drug, which is expected to be marketed under the brand name Alhemo after final approval from the European Commission, will be the first once-daily subcutaneous prophylactic treatment for hemophilia in patients 12 years of age and older who are accompanied by inhibitors. In hemophilia treatment, patients may develop inhibitors due to the immune response, limiting treatment options. Alhemo is unique in that it promotes the formation of blood clots in the presence of inhibitors in the patient's body. Stephanie Seremetis, Novo Nordisk's chief medical officer for hemophilia, said in a press release that the CHMP recommendation is an "important milestone" for patients, noting that Alhemo will provide hemophiliacs with a new option for daily bleeding prevention while being easy to deliver via a pre-filled portable pen. Concizumab, the lead drug candidate against TFPI, was rejected by the FDA last year. In its response letter, the FDA asked Novo Nordisk to provide more information about the drug's monitoring, patient dosing and manufacturing process. Pfizer's competitors have since gained FDA approval. Novo Nordisk has resubmitted its application for Concizumab to the FDA and it has been approved in several countries. However, the CHMP has again expressed its displeasure with PTC Therapeutics' Translarna drug, the latest in a series of setbacks for Translarna in Europe. The CHMP first rejected Translarna's full approval last September, citing a lack of efficacy, and reached the same conclusion in another review in January. Although the European Commission requested another review in May, the latest negative opinion from the CHMP could leave Translarna's future in Europe in uncertainty. Matthew B. Klein, MD, CEO of PTC Therapeutics, said in a press release that the CHMP's opinion is contrary to the wishes of European physicians, patients and their families. The PTC will continue to ensure that boys and young men with the nonsense mutation Duchenne muscular dystrophy have access to Translarna and will provide the European Commission with all possible evidence to support continued authorization. Citi analysts predicted in the report that the European Commission is likely to accept the CHMP's opinion after its review. Although Translarna received conditional authorization in Europe in 2014, the FDA has rejected applications for the drug twice. PTC Therapeutics said it plans to apply for FDA approval again this year.

Pfizer’s anti-tissue factor pathway inhibitor Hympavzi (marstacimab-hncq) has been approved by the US Food and Drug Administration (FDA) to treat haemophilia A or B in adult and paediatric patients aged 12 years and older. Hympavzi, which is now the first in its drug class to be approved in the US for haemophilia A or B, has been specifically authorised for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients with haemophilia A without factor VIII inhibitors or haemophilia B without factor IX inhibitors. More than 800,000 people globally are affected by haemophilia, a family of rare genetic blood disorders caused by a clotting factor deficiency. Despite recent progress in haemophilia treatment, many patients continue to experience repeated bleeding episodes and manage their condition with frequent intravenous infusions that may need to be given multiple times a week. Administered as a once-weekly subcutaneous injection, Hympavzi works by reducing the amount of the naturally occurring anticoagulation protein, tissue factor pathway inhibitor. This increases the amount of thrombin, an enzyme critical in blood clotting, that is generated. The FDA’s decision was supported by positive results from the late-stage BASIS trial, in which Hympavzi reduced the annualised bleeding rate for treated bleeds by 35% and 92% after a 12-month active treatment period compared to routine prophylaxis and on-demand treatment, respectively, in patients with haemophilia A or B without inhibitors. Ann Farrell, director of the division of non-malignant haematology in the FDA’s Center for Drug Evaluation and Research, said the approval “provides patients with haemophilia a new treatment option that is the first of its kind to work by targeting a protein in the blood clotting process”. The authorisation comes less than six months after the FDA approved Pfizer’s one-time haemophilia B gene therapy Beqvez (fidanacogene elaparvovec-dzkt) for use in certain adults with moderate-to-severe cases of the disorder. The company also has a range of recombinant factor treatments. Aamir Malik, Pfizer’s chief US commercial officer and executive vice president, said: “We look forward to launching this latest medical breakthrough and to now offer three distinct classes of haemophilia medicines… that can meet the unique treatment needs of a wide range of patients.”

Pictured: Pfizer office at its South San Francisco iStock, hapabapa The approval makes Pfizer’s Hympavzi the first once-weekly subcutaneous prophylactic injection for hemophilia B in the U.S., according to the company, which is currently embroiled in a row with activist investor Starboard Value. The FDA on Friday signed off on Pfizer ’s marstacimab-hncq for the treatment of hemophilia A or hemophilia B in adults or adolescents, which will now be marketed under the brand name Hympavzi. Friday’s approval makes Hympavzi the first once-weekly subcutaneous prophylactic injection for hemophilia B in the U.S., according to Pfizer’s announcement. Hympavzi is also the first treatment available through a pre-filled pen or syringe for both hemophilia A and hemophilia B. According to its label , Hympavzi is indicated for routine prophylaxis to lower the frequency or completely prevent bleeding episodes in hemophilia patients. The treatment is administered once a week via subcutaneous injection and is approved for hemophilia A patients without factor VIII inhibitors or hemophilia B patients without factor IX inhibitors. Suchitra Acharya, program head of the Bleeding Disorders and Thrombosis Program at Cohen Children’s Medical Center, in a statement called Hympavzi’s approval a “meaningful advancement for people living with hemophilia A or B,” many of whom continue to suffer from persistent bleeding and experience difficulties in managing their conditions despite progress in the field. “Hympavzi aims to reduce the current treatment burden by meeting an important need for these patients, including many who have required frequent, time-consuming intravenous treatment infusion regimens,” according to Acharya, who previously disclosed receiving honoraria from Pfizer. The approval was backed by data from the Phase III BASIS trial, an open-label study that enrolled nearly 190 patients with severe hemophilia A or moderately severe to severe hemophilia B. Pfizer posted data from BASIS in May 2023, touting a 92% reduction in bleeding in patients lacking inhibitors to factor VIII or factor IX. Annual bleeding rate dropped by 35%. Hympavzi is an IgG1 monoclonal antibody that targets the Kunitz domain 2 of the tissue factor pathway inhibitor protein, which under healthy circumstances prevents blood clotting. This mechanism of action allows Hympavzi to enhance coagulation in hemophilia patients. Friday’s regulatory win bolsters Pfizer’s hemophilia portfolio, anchored by the gene therapy Beqvez, which won the FDA’s green light in April 2024 for moderate to severe hemophilia B. The company is also developing another gene therapy, giroctocogene fitelparvovec, for hemophilia A. Phase III data released in July 2024 showed that the investigational treatment was both non-inferior and superior to regular Factor VIII infusions. Hympavzi’s approval comes as Pfizer continues to grapple with activist investor Starboard Value which last week took a $1 billion stake in the pharma , hoping to depose CEO Albert Bourla and shake up the direction of the company. However, former Pfizer CEO Ian Read and former CFO Frank D’Amelio reaffirmed their support for the company’s current leadership. Starboard Value in a letter to Pfizer’s board claimed Read and D’Amelio were pressured to issuing a statement of support for Bourla and senior management.