PFE - FDA Green Lights Pfizer's Gene Therapy For Rare Bleeding Disorder Hemophilia | Benzinga
Friday, the FDA approved Pfizer Inc’s (NYSE:PFE) Beqvez (fidanacogene elaparvovec-dzkt) for moderate to severe hemophilia B in adult patients who currently use factor IX (FIX) prophylaxis therapy or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var capsid.
Beqvez is a one-time treatment designed to enable hemophilia B patients 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 ...