In February 2021, the U.S. Food and Drug Administration (FDA) partially rescinded a supplemental Biologics License Application (BLA) that the agency had granted, in error, for the recombinant factor IX product BeneFIX® (Pfizer). The temporary rescission centered on a specific indication for routine prophylaxis to reduce the frequency of bleeding episodes in children (<16 years of age) with hemophilia B. It had been granted by mistake, as another product already held orphan drug exclusivity until September 12th of this year.
As of September 13th, this indication for BenFIX® has been fully restored by the FDA. The product’s full set of indications now includes adults and children with hemophilia B (congenital factor IX deficiency or Christmas disease) for: on-demand treatment and control of bleeding episodes; perioperative management of bleeding; routine prophylaxis to reduce the frequency of bleeding episodes.
To see a complete list of products, go to Medical and Scientific Advisory Council (MASAC) Document 263 – MASAC Recommendations Concerning Products Licensed for the Treatment of Hemophilia and Other Bleeding Disorders.