FDA Approves Beqvez (fidanacogene elaparvovec-dzkt) One-Time Gene Therapy for Adults with Hemophilia B
Beqvez™, manufactured by Pfizer for the treatment of adults with moderate to severe hemophilia B, has been approved by the FDA for patients with the following:
o Currently use factor IX (FIX) prophylaxis therapy, or
o Have current or historical life-threatening hemorrhage, or
o Have repeated, serious spontaneous bleeding episodes, and
o Do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test.
- Beqvez is a one-time treatment that is designed to enable people living with hemophilia B to produce FIX themselves rather than the current standard of care, which requires regular intravenous infusions of FIX that are often administered multiple times a week or multiple times a month.
- Hemophilia B is a rare genetic bleeding disorder that prevents normal blood clotting because of a deficiency in FIX that causes those with the disease to bleed more frequently and longer than others.
- According to the World Federation of Hemophilia, more than 38,000 people worldwide are living with hemophilia B.
