Pfizer's fidanacogene elaparvovec accepted for review by FDA and EMA

The US Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for fidanacogene elaparvovec for the treatment of adults with hemophilia B, said US pharma giant Pfizer (NYSE: PFE).

In parallel, the European marketing authorization application (MAA) for fidanacogene elaparvovec has also been accepted and is under review by the European Medicines Agency (EMA).

Fidanacogene elaparvovec, which Pfizer is developing with Swiss pharma giant Roche (ROG: SIX), is a novel, investigational gene therapy that contains a bio-engineered adeno-associated virus (AAV) capsid (protein shell) and a high-activity variant of human coagulation Factor IX (FIX) gene. For people living with hemophilia B, the goal of this gene therapy is to enable them to produce FIX themselves via this one-time treatment rather than needing regular intravenous infusions of FIX, as is the current standard of care.