Beroctocog alfa

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%.

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.

DelveInsight's 'Hemophilia A Pipeline Insight 2024' covers global Hemophilia A therapies, highlighting over 40+ pipeline therapies, key companies like Spark Therapeutics, Pfizer, and Sanofi, and recent FDA approvals for Roctavian and ALTUVIIIO. The report assesses the therapeutic landscape, including product types, stages, routes of administration, and molecule types.

DelveInsight's 'Hemophilia A Pipeline Insight 2024' covers global Hemophilia A therapies, highlighting over 40+ pipeline therapies, key companies like Spark Therapeutics, Pfizer, and Novo Nordisk, and recent FDA approvals for Roctavian and ALTUVIIIO.

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.