FDA Approves Gene Therapy for Hemophilia B

Click here for CSL Behring’s press release from November 22, 2022, announcing FDA approval of etranacogene dezaparvovec [Hemgenix] for adult Hemophilia B [factor IX deficient] patients.

This is the culmination of 20 years of research led by uniQure LLC and may usher in gene transfer therapy for additional ‘monogenic’ disorders in the future such as Hemophilia A [factor VIII deficiency]. Thanks to Precision BioLogic colleague and friend Dean Willett for the heads-up. Click here for a “Fierce Pharma” editorial describing the Hemgenix fee structure and click the citation below to read about the HOPE-B trial whose data support the efficacy and safety of Hemgenix: Von Drygalski A, Giermasz A, Castaman G, et al. Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B. Blood Adv. 2019 12;3(21):3241-3247. doi: 10.1182/bloodadvances.2019000811.