CSL Reports Temporary Shortage of Hemophilia B Gene Therapy

Biopharmaceutical company CSL Limited says it is experiencing a temporary global shortage of its gene therapy Hemgenix, potentially delaying treatment for some patients with hemophilia B. In a letter addressed to the hemophilia B community, the company says the shortage is not related to any concerns about the therapy’s safety or effectiveness. Instead, CSL attributed the supply disruption to the inherent complexity of manufacturing gene therapies and its adherence to stringent regulatory and quality standards.

Hemgenix is a one-time gene therapy designed for adults with hemophilia B who are on Factor IX prophylaxis or have a history of severe or life-threatening bleeding. Administered as a single intravenous infusion, the therapy aims to enable the body to produce Factor IX, potentially reducing or eliminating the need for ongoing treatment.