Roctavian (valoctocogene roxaparvovec-rvox) was approved June 29 to reduce bleeding in adults patients with the blood disorder, according to a news release from the San Rafael, Calif.-based drugmaker.
Every year, hemophilia A affects 1 in 5,000 male births, according to the CDC. Because of the rarity of the disorder, the approval was based on a study of about 130 patients. In the trial, the drug reduced annualized bleeding rate in hemophilia A patients, on average, by 52 percent.
The drug’s cost is $2.9 million.
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