Managed Healthcare Executive April 27, 2024
This week, the FDA has approved several products, including Beqvez, the second gene therapy hemophilia B; Ojemda for children with brain tumors; an additional indication for Lutathera for adolescents with neuroendocrine tumors; and a bladder cancer immunotherapy. The agency has also issued a complete response letter for pz-cel to treat epidermolysis bullosa.
FDA Approves Pfizer’s Gene Therapy for Beqvez for Hemophilia B
The FDA has approved Pfizer’s one-time gene therapy Beqvez (fidanacogene elaparvovec-dzkt) to treat adults with moderate-to-severe hemophilia B. It is indicated for those who currently use factor IX (FIX) prophylaxis therapy, have current hemorrhage, repeated bleeding episode or do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid.
Hemophilia is a rare genetic bleeding disorder caused...
Today's Sponsors
Today's Sponsor