Rising Adoption of Gene Therapy and Extended Half-Life Products”
- A significant and accelerating trend in the global hemophilia market is the increasing adoption of gene therapies and extended half-life (EHL) factor replacement therapies, which aim to reduce treatment frequency and improve quality of life for patients. These advanced therapies are transforming the standard of care, offering sustained benefits with fewer infusions
- For instance, in November 2022, CSL Behring’s Hemgenix received FDA approval as the first gene therapy for hemophilia B, delivering a one-time treatment that significantly reduces annual bleeding rates. Similarly, BioMarin’s Roctavian gene therapy for hemophilia A gained EU approval, with U.S. approval following in 2023
- EHL products such as Elocta (Sobi), Adynovate (Takeda), and Alprolix (Sobi) are designed to maintain clotting factor levels longer, minimizing the frequency of infusions required. These therapies offer more consistent protection from bleeding and are increasingly favored by patients and physicians
- The shift towards gene therapies is also driven by their potential to offer long-term or even curative outcomes, which could significantly reduce the lifetime cost and burden of traditional prophylaxis. However, due to their novelty and high cost, uptake is currently concentrated in developed markets with advanced reimbursement frameworks
- This trend towards innovative, long-acting, and potentially curative treatments is reshaping patient management and fueling R&D investments. Consequently, companies such as Pfizer, Spark Therapeutics, and Sangamo Therapeutics are actively developing next-generation gene therapies targeting both hemophilia A and B
- The demand for safer, more convenient, and longer-lasting therapies is growing rapidly across the global hemophilia community, with healthcare systems gradually adapting to support these advancements in clinical practice and reimbursement
