CSL reports a temporary global stockout of Hemgenix gene therapy for hemophilia B due to manufacturing complexities, with no impact on the therapy’s safety or effectiveness.
Written By: Sana Khan BPharm
Reviewed By: Pharmacally Editorial Team
CSL has announced a temporary global stockout of HEMGENIX® (Etranacogene Dezaparvovec-drlb), the company’s one-time gene therapy for hemophilia B, which may delay treatment for some eligible patients in countries where the therapy is commercially available.
According to the company, the supply interruption is not related to the safety or effectiveness of Hemgenix. Instead, it reflects the complexities involved in manufacturing advanced gene therapies and the company’s commitment to maintaining strict regulatory and quality standards.
CSL stated that it is actively working with regulatory authorities on strategies to stabilize supply while preserving the high manufacturing standards required for gene therapy products. The company acknowledged that the situation may raise concerns among patients and healthcare providers and emphasized that ensuring access to accurate information and maintaining confidence in the treatment remain top priorities.
Hemgenix is a one-time adeno-associated virus (AAV) gene therapy designed to treat adults with hemophilia B by enabling the body to produce functional factor IX, potentially reducing or eliminating the need for routine factor replacement therapy.
Dr. Deborah Long, Senior Vice President of Medical Affairs at CSL, said the company remains fully committed to delivering the therapy to the hemophilia B community and will continue to provide updates as efforts to ensure stable supply progress.
CSL also noted that it recognizes the careful planning involved in gene therapy decisions and reaffirmed its long-standing partnership with the hemophilia B community, pledging continued communication to support patients and healthcare providers in managing treatment plans.
References
Our Commitment to the Hemophilia B Community: An Update on HEMGENIX® (Etranacogene Dezaparvovec-drlb) Availability, 17 March 2026, News Releases | CSL
About the Writer
Sana Jamil Khan is a B.Pharm graduate with a strong interest in medical writing and scientific communication. Her work focuses on interpreting clinical research, exploring developments in pharmaceutical science, and presenting complex medical information in a clear and accessible manner. She is particularly interested in topics related to human clinical studies, drug safety observations, and emerging therapeutic research.