Pfizer scores FDA nod for hemophilia B gene therapy, will charge $3.5M per dose
Pfizer has gained FDA approval for Beqvez, a gene therapy treatment for adults with hemophilia B, a bleeding disorder. The FDA has endorsed the treatment, making it the first FDA-approved gene therapy for Pfizer and the second in the indication after CSL and uniQure's Hemgenix. Pfizer decided to charge the same $3.5 million for Beqvez, which will be available to patients this quarter. Hemgenix, the world's most expensive drug, was approved in 2022 at $3.5 million. The current number of males with hemophilia in the U.S. is around 33,000, with Hemophilia A being more prevalent. Last month, Kyowa Kirin and Orchard Therapeutics placed a $4.25-million tag on Lenmeldy, their gene therapy for metachromatic leukodystrophy (MLD). Pfizer believes that Beqvez presents a compelling value proposition that can deliver savings for the healthcare system when considering potential long-term benefits that would offset costs associated with chronic disease management. The therapy can free patients from standard-of-care intravenous transfusions, which can cost up to $600,000 and up to $1.1 million per year. Pfizer is offering a warranty program to provide financial protections by insuring against the risk of efficacy failure.
