US FDA approves Pfizer’s gene therapy for rare bleeding disorder

By Thomson Reuters Apr 26, 2024 | 5:54 AM

(Reuters) – The U.S. Food and Drug Administration approved Pfizer’s gene therapy for hemophilia B on Friday, the second such therapy for the rare bleeding disorder that typically requires regular infusions of a blood-clotting protein.

People with hemophilia have a fault in a gene that regulates production of proteins called clotting factors, which can cause spontaneous as well as severe bleeding following injuries or surgery. It predominately affects males.

(Reporting by Sriparna Roy in Bengaluru; Editing by Shailesh Kuber)