Fidanacogene Elaparvovec, a one-shot hemophilia treatment, has been designated an orphan drug. The Ministry of Food and Drug Safety recently announced that it has selected Fidanacogene Elaparvovec, Pfizer's hemophilia B gene therapy, as an orphan drug. Fidanacogene Elaparvovec is a method that combines adenoviral vector (AVV) capsid and highly active coagulation factor IX gene and is characterized by producing coagulation factor IX in one cycle instead of regular injection am.

The drug has been designated as a Breakthrough Therapy, Advanced Regenerative Medicine and Therapeutics (RMAT), and Orphan Drug by the US FDA, and PRIority MEdicines and Orphan Drug by the European EMA. The phase 3 BENEGENE-2 stud