Beroctocog alfa

In 2024, Hemophilia News Today highlighted key advancements in hemophilia treatment, including gene therapies like BBM-H803 for hemophilia A, Mim8's efficacy, and the launch of Beqvez for hemophilia B. Roctavian showed significant bleeding reduction, and the FDA cleared a CRISPR therapy trial for hemophilia B, marking a year of progress in hemophilia care.

The FDA approved Novo Nordisk’s Alhemo, a daily subcutaneous treatment for hemophilia A or B patients aged 12+ with inhibitors, reducing bleeding episodes. It’s the first of its kind, available in prefilled pens, and shown to significantly decrease bleeds in trials.

Pfizer terminated its hemophilia A gene therapy partnership with Sangamo Therapeutics, causing Sangamo's stock to drop 56%. Despite a successful Phase III trial for giroctocogene fitelparvovec, Pfizer decided against seeking approval, citing a smaller-than-expected market. Sangamo plans to explore options for the therapy's future.

Pfizer ends its seven-year partnership with Sangamo Therapeutics on a hemophilia A gene therapy, avoiding costly milestone payments. Sangamo regains rights to the therapy, aiming to find a new partner for regulatory review and commercialization amidst financial challenges.

Biosimilars, offering 20-25% cost reduction over biologics, are set to expand healthcare access. With biologics worth $63 billion going off-patent by 2016, biosimilars' market is expected to grow significantly. Regulatory pathways in the EU and US are evolving, with monoclonal antibodies being the next focus. Emerging markets show potential in biosimilar development, despite quality and regulatory challenges.

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%.

Roche discontinues SPK-8011, a hemophilia A gene therapy, shifting focus to an enhanced FVIII variant for unmet needs. The decision follows SPK-8011's phase 3 study withdrawal, with no patients enrolled. Roche's gene therapy pipeline faces challenges, including previous candidate removals and a new $1B+ deal with Dyno Therapeutics for advanced gene therapy vectors.

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.

Indian scientists successfully treated severe haemophilia A with gene therapy, eliminating bleeding episodes in five patients over 14 months. This one-time treatment, led by CSCR and supported by the Department of Biotechnology, introduces a gene to produce clotting factor, offering a safer, cost-effective alternative to frequent injections. The study, published in NEJM, is hailed as ground-breaking for its potential in resource-constrained settings.

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.