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 […]
