June 9, 2023

FDA approves first gene therapy for hemophilia B

On November 22, 2022, the FDA approved Hemgenix®, an adeno-associated virus vector-based gene therapy indicated for the treatment of adults with Hemophilia B (congenital Factor IX deficiency) who:

  • Currently use Factor IX (FIX) prophylaxis therapy, or
  • Have current or historical life-threatening hemorrhage, or
  • Have repeated, serious spontaneous bleed episodes

The HOC has individuals who expressed interest in Hemgenix, have completed their pre-screening eligibility and are in the final stages of meeting all the requirements needed for gene therapy The HOC medical team has participated in extensive educational trainings and are knowledgeable about all aspects of gene therapy with Hemgenix®. This includes, but is not limited to, preparation for treatment, administration of the therapy, and close monitoring once it has been given.

If you have any questions or want more information about Hemgenix®, please contact your hemophilia treatment center.

LEARN MORE ABOUT HEMGENIX®

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