From CNBC: 2024-04-26 07:19:04

The FDA approved Pfizer’s gene therapy for hemophilia B, Beqvez, targeting adults with the rare bleeding disorder. The treatment, priced at $3.5 million, offers a one-time solution to help patients produce factor IX and prevent bleeding episodes. Pfizer aims to boost its business with transformative gene therapies amidst the decline of its Covid business.

Pfizer’s approval marks a significant milestone in the gene therapy field, focusing on targeted, high-cost treatments. Beqvez will compete with CSL Behring’s Hemgenix, offering a one-time, costly solution to hemophilia B. However, experts note challenges in uptake due to costs and logistical hurdles for similar gene therapies in the market. Pfizer continues to pursue FDA approval for marstacimab to treat hemophilia A and B, as well as gene therapy for Duchenne muscular dystrophy.



Read more at CNBC: FDA approves Pfizer gene therapy Beqvez for treatment of hemophilia B