Marstacimab

Pfizer's phase 3 BENEGENE-2 study showed fidanacogene elaparvovec, a gene therapy for haemophilia B, significantly reduced bleeding rates in males, offering a potential one-time treatment alternative to regular Factor IX infusions. The therapy demonstrated a 71% reduction in annualised bleeding rate and was well-tolerated.

At ASH 2024, physicians discussed advancements in hemophilia care, including Roche's Hemlibra and curative AAV gene therapies like CSL Behring's Hemgenix, Biomarin's Roctavian, and Pfizer's Beqvez and Hympavzi. Despite progress, unmet needs remain, with concerns over long-term risks of gene therapies and limited access. Pfizer presented Phase III AFFINE trial data for giroctocogene fitelparvovec, showing comparable efficacy to Roctavian with better safety. Sanofi's fitusiran, a siRNA, demonstrated efficacy similar to clotting factor replacement therapy. Staidson's bemiltenase alfa, a fusion protein, showed favorable bleed clearance rates. Challenges persist, but advancements in curative, prophylaxis, and on-demand treatments offer more options for patients.

Annette von Drygalski discusses advancements in hemophilia care at the 2024 ASH Annual Meeting, highlighting improved prognosis due to novel therapies and imaging techniques. She outlines the evolution of care, focusing on mechanisms of bleeding, prophylactic treatments, and emerging gene therapies. Despite progress, hemophilic arthropathy remains a challenge, necessitating further understanding and optimization of imaging tools for early detection and management.

Hemophilia B market to grow significantly by 2034, driven by gene therapies like HEMGENIX and BEQVEZ, priced at $3.5 million each. Key companies include Centessa Pharmaceuticals, Sanofi, Pfizer, and Novo Nordisk. Market dynamics influenced by increasing prevalence, prophylactic treatment focus, and pipeline advancements.

Novartis raised mid-term sales guidance, Pfizer's Hympavzi approved in EU for hemophilia, AstraZeneca's Tagrisso recommended for EGFR-mutated lung cancer, J&J and Lilly report positive study data.

Novo Nordisk's Alhemo (concizumab) recommended for approval by EMA's CHMP as first once-daily, subcutaneous prophylactic treatment for hemophilia A or B with inhibitors in EU. Alhemo targets TFPI to aid blood clotting, potentially alleviating treatment burden with a portable, room-temperature-stable pen. Final EC approval expected by mid-to-late December 2024.

Novo Nordisk's Alhemo (concizumab) recommended for approval by EMA's CHMP as first once-daily, subcutaneous prophylactic treatment for hemophilia A or B with inhibitors in persons 12 years or older. Alhemo targets TFPI to aid blood clotting and reduce bleeding in hemophilia patients with inhibitors.

Novo Nordisk's concizumab (Alhemo) recommended for EU approval as first once-daily subcutaneous prophylactic treatment for haemophilia A/B with inhibitors. Already approved in Japan, Australia, and Switzerland, but FDA rejected in 2022. Competes with Pfizer's Hympavzi and Roche's Hemlibra, offering potential for everyday prophylaxis to prevent bleeds.

FDA approves Hympavzi (marstacimab) for hemophilia, targeting blood-clotting proteins to prevent bleeding in patients aged 12+ without inhibitors to factor VIII or IX.

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.