Coagulation Factor IX Human

At the 66th ASH Annual Meeting, Steven W. Pipe, MD, presented ATLAS-OLE trial data on fitusiran, an siRNA therapeutic targeting antithrombin, showing its effectiveness as a prophylactic agent for hemophilia A and B, reducing breakthrough bleeds by 70% and factor consumption by 64-98%.

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.

Be Biopharma announces updates at ASH Annual Meeting, including BeCoMe-9 trial poster on BE-101, a BCM for Hemophilia B, and an oral presentation on BCM platform versatility in genetic disease and oncology.

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.

The FDA approved Pfizer’s HYMPAVZI (marstacimab-hncq) for routine prophylaxis in hemophilia A or B without inhibitors, administered via a pre-filled auto-injector pen with a once-weekly dosing schedule. HYMPAVZI is the first anti-TFPI approved for hemophilia treatment in the U.S. and aims to reduce treatment burden with a manageable safety profile.

FDA approves Pfizer's Hympavzi, the first anti-TFPI for hemophilia A or B, administered via an auto-injector pen, reducing bleeding episodes in patients. Expected sales of $300 million by 2030.

Pfizer's Hympavzi, an anti-TFPI approved by the FDA, offers a once-weekly subcutaneous treatment for hemophilia A or B without inhibitors, reducing bleeding episodes and treatment burden.

Pfizer's Hympavzi, a once-weekly injection for hemophilia A or B, received FDA approval. The drug, administered via auto-injector, aims to reduce bleeding episodes and treatment burden. Sales are projected to reach $300 million by 2030.

Pfizer's Hympavzi, a once-a-week injection, received FDA approval for treating haemophilia A or B in patients aged 12 and older. The drug aims to reduce treatment burden and showed a 92% reduction in bleeding episodes in a late-stage study. Hympavzi is Pfizer's second haemophilia treatment approved this year, with potential sales of $300 million by 2030.