Gene Therapy for Hemophilia
In November, the FDA approved the first treatment of its kind for hemophilia B, a rare genetic condition that saps a person’s ability to properly clot blood. The treatment, now called Hemgenix, is a gene therapy that delivers a functional copy of the gene that’s dysfunctional in those with hemophilia B.
Developed by CSL Behring, Hemgenix won’t come cheap. It’s expected to cost $3.5 million per patient, the highest list price yet for a single-dose treatment. But its makers expect the drug to be worthwhile, since it should reduce how often patients need expensive infusions of their missing clotting factor. Other gene therapies for hemophilia A and B may soon reach the market as well.
