Skip to main content
Premium Trial:

Request an Annual Quote

A High Price

A gene therapy for hemophilia could cost millions of dollars, according to NPR.

It reports that BioMarin Pharmaceutical is seeking regulatory approval for its hemophilia gene therapy from authorities in the US and Europe. Earlier this year, researchers led by BioMarin's Wing Wong reported in the New England Journal of Medicine that a small group of patients with severe hemophilia A who received the treatment had fewer bleeding events.

According to NPR, BioMarin has said the treatment could run $3 million. Jeff Ajer, BioMarin's executive vice president and chief commercial officer, tells it that hemophilia is an expensive disease to treat and if their approach is, as hoped, a one-time treatment, it could actually save money.

But, NPR notes that Memorial Sloan Kettering Cancer Center's Peter Bach, who studies drug pricing, calls this "just outrageous." The Hemophilia Federation of America's Meg Bradbury adds that "[w]e need to make sure all those who are eligible would have access to" the treatment.

Ajer notes at NPR that the firm is working with insurance companies and government programs to cover the costs.

Other gene therapies have also come with high price tags. For instance, Spark Therapeutics' Luxturna, which treats retinal dystrophy, costs $425,000 per eye; Novartis' CAR-T therapy Kymriah for acute lymphoblastic leukemia costs $475,000; and Kite Pharmaceuticals' Yescarta for large B-cell lymphoma costs $373,000. Novartis' Zolgensma, a one-time treatment for spinal muscular atrophy, costs $2.1 million.