According to an updated post on the Center for Biologics Evaluation and Research (CBER) webpage, there were twelve Biologics License Applications (BLAs) approved for products that had orphan drug (OD) status. Just to cite a few:

  • Two were for treatment of sickle cell disease (SCD).
  • One was for enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP).
  • One was for the urgent reversal of acquired coagulation factor deficiency induced by Vitamin K antagonist (VKA, e.g., warfarin), along with one BLA for another bleeding disorder.

The number of OD approvals in 2022 was one-third (4) of the number approved in 2023. This could signal a trend in BLA approvals for OD indications for rare diseases.

The full list can be found here.