Move over, Zolgensma. A more expensive multimillion dollar drug could soon be on the market.
Last year, after the FDA approved Zolgensma — a gene therapy treatment for spinal muscular atrophy sold by Novartis— its $2.1 million price tag made it the world’s most expensive drug. Now, another gene therapy developed by BioMarin Pharmaceuticals could soon take Zolgensma’s top spot. Called Valrox, the one-and-done treatment for hemophilia A, which prevents patients from forming blood clots, could come with a $3 million price tag.
The arguments around the price are familiar to developers of gene therapies. While some drug pricing watchdogs call the cost “outrageous,” BioMarin argues that because it currently costs hundreds of thousands of dollars a year to treat patients with hemophilia A, who typically endure infusions of a blood-clotting factor two or three times a week, the treatment could save millions in the long-run.
In one early study, the therapy cut bleeding episodes in all of the 13 patients (the condition is rare). A later-stage study of 150 patients is currently underway and BioMarin has reported that so far, the results have been positive.
The FDA granted Valrox a “breakthrough therapy” designation and is expected to render a final approval decision by Aug. 21.
