Marstacimab

FDA approves Hympavzi (marstacimab, Pfizer) for routine prophylaxis in hemophilia A/B patients without factor inhibitors, targeting TFPI to increase thrombin. Hympavzi is the first non-factor, once-weekly treatment for hemophilia B in the US, reducing bleeding episodes in BASIS trial participants.

FDA approves Hympavzi, a new preventive treatment for hemophilia A or B, reducing bleeding episodes to a few per year. Hympavzi, a once-per-week shot, targets a protein in the blood clotting process, offering an alternative to frequent infusions. Clinical trials showed significant reduction in bleeding episodes, with side effects including needle site reactions, headache, and itching.

Pfizer’s anti-TFPI antibody marstacimab (Hympavzi) approved by FDA for treating haemophilia A and B without inhibitors, offering once-weekly subcutaneous dosing via pen-injector. Competes with Roche’s Hemlibra and Sanofi’s Altuviiio, with potential in haemophilia B over standard clotting factor therapies. Achieved 35% reduction in annualised bleeding rate (ABR) in BASIS trial. Priced at $795,600 per year, pending EU approval.

The FDA approved marstacimab (Hympavzi) for routine prophylaxis to prevent bleeding in hemophilia A or B patients without inhibitors. It targets the TFPI protein, reducing bleeding rates significantly in clinical trials. The drug offers a once-weekly subcutaneous administration, easing treatment burden.

The FDA approved Pfizer’s Hympavzi for routine prophylaxis in hemophilia A or B without inhibitors, administered via a prefilled autoinjector pen. Hympavzi is the first once-weekly subcutaneous injection therapy for hemophilia B in the U.S., designed to reduce bleeding episodes by binding and blocking the activity of tissue factor pathway inhibitor (TFPI). The therapy aims to reduce treatment burden and is under review by the European Medicines Agency for potential approval.

FDA approves marstacimab, an anti-TFPI for hemophilia A and B, reducing bleeding episodes. Hympavzi, by Pfizer, is the first subcutaneous option for hemophilia B, administered weekly via autoinjector. The approval is based on the phase 3 BASIS trial, showing significant reduction in annualized bleeding rates and manageable safety profile.

EMA's CHMP recommends approval of marstacimab in EU for preventing bleeding in severe hemophilia A or B without inhibitors. European Commission to review, with final decision expected soon. If approved, Pfizer's treatment, Hympavzi, will be available as a 150 mg subcutaneous injection. CHMP's positive opinion supported by Phase 3 BASIS trial data showing marstacimab significantly reduced bleed rates in patients without inhibitors.

EMA's CHMP recommended approval for 8 new medicines and extended indications for 12 others, including treatments for cancers, hemophilia, meningococcus, and biosimilars for age-related macular degeneration. Updates to COVID-19 vaccines Spikevax and Comirnaty targeting new variants were also recommended. CHMP extended indications for mpox vaccine Imvanex and others, while refusing authorization for Syfovre. Bruno Sepodes was elected as new CHMP chair, prioritizing medicine quality, safety, and efficacy.

EMA's CHMP recommended approval for 8 new medicines and extended indications for 12 drugs, including treatments for cancers, hemophilia, meningococcus, and biosimilars for age-related macular degeneration. CHMP also updated COVID-19 vaccines Spikevax and Comirnaty to target new variants. Bruno Sepodes was elected as the new CHMP chair, prioritizing medicine quality, safety, and efficacy.

The Committee for Human Medicinal Products (CHMP) elected Bruno Sepodes as its new chair, effective 21 September. CHMP recommended marketing authorisations for eight new medicines, including Elahere, Hetronifly, Hympavzi, Penbraya, Theralugand, Afqlir, Opuviz, and Pomalidomide Teva. It also recommended extending indications for 12 existing medicines and confirmed the refusal of Syfovre's marketing authorisation. Applications for Durysta, Tecentriq, and Sialanar were withdrawn. CHMP updated Spikevax and Comirnaty vaccines to target new COVID-19 variants.