The Food and Drug Administration approved Pfizer's hemophilia B gene therapy on Friday, a one-time treatment that will cost $3.5 million in the U.S.
Pfizer's drug, dubbed Beqvez, will rival another gene therapy called Hemgenix from CSL Behring and Uniqure. The duo gained FDA approval in late 2022. Hemgenix has the same price tag and trails Orchard Therapeutics' gene therapy, Lenmeldy, in terms of price. Lenmeldy won its U.S. approval last month for a rare genetic condition and goes for $4.25 million a dose.
Patients with hemophilia B lack a key protein that helps the blood clot and often experience severe bleeding episodes. Generally, patients must receive monthly infusions of Factor IX, the missing protein. But Pfizer says Beqvez is a one-time treatment that will help patients' bodies make their own Factor IX, eliminating the need for regular infusions.
"A one-time treatment with Beqvez has the potential to be transformative for appropriate patients by reducing both the medical and treatment burden over the long term," Adam Cuker said in a statement. Cuker is the director of the Penn Comprehensive and Hemophilia Thrombosis Program.
Pfizer stock rose a fraction to close at 25.40 on Friday. Shares are now trading at their lowest point in 11 years.
Pfizer Stock: A New Hemophilia B Gene Therapy
Beqvez is approved for adults with moderate to severe hemophilia B.
The gene therapy is made using an adeno-associated viral vector. In essence, this technology uses a harmless virus to deliver the drug. Patients must undergo a genetic test before treatment to confirm they don't have preexisting antibodies to the delivery virus.
The case is the same for Hemgenix, the CSL and Uniqure gene therapy. Hemgenix was the most expensive drug in the U.S. at $3.5 million a dose when it gained approval in 2022. Last month, Orchard topped that price tag with the Lenmeldy approval. Lenmeldy treats metachromatic leukodystrophy, a rare condition in which the protective fatty layer surrounding nerves begins to fall apart.
Pfizer is also working on a hemophilia A and B treatment called marstacimab and a gene therapy for hemophilia A called giroctocogene fitelparvovec. The second gene therapy is in final-phase testing. Hemophilia A is tied to lacking Factor VIII, another clotting protein.
Pfizer stock remains under pressure, however. Shares are below their 50-day and 200-day moving averages, according to MarketSurge.com.
This story has been corrected to reflect Lenmeldy is now the most expensive drug in the U.S.
Follow Allison Gatlin on X, the platform formerly known as Twitter, at @IBD_AGatlin.
