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Two New Gene Therapies Granted FDA Approval

On June 22, 2023, the FDA granted approval to Sarepta Therapeutics’ Elevidys (delandistrogene moxeparvovec-rokl), this is the first gene therapy for the treatment of patients 4 through 5 years of age with Duchenne Muscular Dystrophy (DMD) with a confirmed mutation in the DMD gene. Sarepta announced the pricing for Elevidys at $3.2 million for the one-time treatment. On June 29, 2023, the FDA also approved BioMarin Pharmaceutical’s Roctavian (valoctocogene roxaparvovec-rvox), the first gene therapy for the treatment of adults with severe hemophilia A. BioMarin stated that Roctavian will be commercially available in 2 months at a wholesale acquisition cost (WAC) of $2.9 million for the one-time treatment.

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