FDA approval of BioMarin’s Roctavian to test gene therapy pricing model

FDA’s approval of Roctavian from BioMarin to treat severe hemophilia A marks another 2023 gene therapy milestone, and the launch will serve as an important test of the market for gene therapies with possible efficacy expiration dates.

BioMarin Pharmaceuticals Inc. (NASDAQ:BMRN) is launching Roctavian valoctocogene roxaparvovec at a wholesale acquisition cost of $2.9 million and a promise to provide an outcomes-based warranty to all government and commercial payers in the U.S. that will reimburse up to 100% of the cost if a person does not respond. ...