Pfizer Gets FDA OK For Beqvez Gene Therapy for Hemophilia B

By Colin Kellaher

Pfizer has won Food and Drug Administration approval of Beqvez, a one-time gene therapy for certain patients with the inherited bleeding disorder hemophilia B.

The New York drugmaker on Friday said the approval covers the treatment of adults with moderate to severe hemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and don't have neutralizing antibodies to adeno-associated virus serotype Rh74var.

Hemophilia B is a rare disorder that prevents normal blood clotting because of a deficiency in factor X that causes those with the disease to bleed more frequently and longer than others.

Pfizer said Beqvez is designed to enable people living with hemophilia B to produce factor IX themselves rather than the current standard of care, which requires regular intravenous infusions that are often administered multiple times a week or month.

Write to Colin Kellaher at [e-mail]

(END) Dow Jones Newswires

04-26-24 0714ET