The Food and Drug Administration has approved a gene therapy treatment for adults with hemophilia B that costs $3.5 million per dose, making it the world’s most expensive medicine.
“Today’s approval provides a new treatment option for patients with hemophilia B and represents important progress,” Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said Tuesday in an announcement.
Biopharmaceutical firm CSL Behring manufactures the gene therapy treatment Hemgenix, which allows patients with hemophilia to produce a protein that makes their blood clot in open wounds and halt prolonged bleeding.
Each dose of Hemgenix costs $3.5 million, but the gene therapy treatment only requires a single dose, according to the FDA.
Some experts see the price as high but acknowledge that existing treatments for hemophilia are also expensive and say the fear of bleeding complications will drive patient demand.
“While the price is a little higher than expected, I do think it has a chance of being successful because 1) existing drugs are also very expensive and 2) hemophilia patients constantly live in fear of bleeds,” biotechnology investor Brad Loncar told Bloomberg.
Advocates for hemophilia B patients were also pleased with the federal approval of the new treatment.
“Gene therapy is the first treatment option to offer those living with hemophilia B — and caregivers — the possibility of freedom from the need for regular, ongoing infusions,” Kim Phelan, chief operating officer of the Coalition for Hemophilia B, said in CSL Behring’s Hemgenix announcement.