FDA approves gene therapy for hemophilia B, now the world’s most expensive drug

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 most expensive drug in the world.

“Today’s approval provides a new treatment option for patients with hemophilia B and represents significant progress,” Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said Tuesday.

Biopharmaceutical company CSL Behring makes the gene therapy treatment Hemgenix, which enables patients with hemophilia to produce a protein that causes their blood to clot in open wounds and stops prolonged bleeding.

Each dose of Hemgenix costs $3.5 million, but gene therapy treatment requires only one dose, according to the FDA.

Some experts consider the price to be high, but acknowledge that existing hemophilia treatments are also expensive and say fear of bleeding complications will drive patient demand.

“Although the price is a bit higher than expected, I think it has a chance of succeeding because 1) existing drugs are also very expensive and 2) hemophilia patients live in constant fear of bleeding,” said biotech investor Brad Loncar to Bloomberg.

Hemophilia B patient advocates were also pleased with federal approval of the new treatment.

“Gene therapy is the first treatment option to offer people with hemophilia B – and caregivers – the ability to eliminate the need for regular, continuous infusions,” said Kim Phelan, chief operating officer of the Hemophilia B Coalition, in CSL Behring. Hemgenix ad.


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