Pfizer's gene therapy Beqvez, designed for hemophilia B patients, receives US approval.

Pfizer's first gene therapy, Beqvez, has received US approval for patients with hemophilia B, a rare bleeding disorder. The gene therapy is a one-time treatment designed to insert a correct copy of the mutated gene that causes hemophilia B, preventing dangerous bleeding episodes. Hemophilia B affects about 40,000 people worldwide. Pfizer's approval follows CSL Behring's gene therapy for hemophilia B, which gained FDA approval in late 2022.

April 26, 2024
8 Articles