BioMarin’s Roctavian Cuts Bleeds, Draws Interest From Payers
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BioMarin Pharmaceutical Inc.’s Roctavian was still effective three years after patients received the one-time treatment for hemophilia A, the company said Sunday while disclosing its first contract with a German insurer for the gene therapy.
Roctavian reduced the annualized rate of bleeding in patients with hemophilia A by 80% in a three-year study. The company said 92% of patients weren’t taking prophylactic treatments three years after they received Roctavian. Patients who received the one-time treatment used 94% fewer replacement infusions of factor VIII, a natural clotting substance that’s missing in people with the disorder, BioMarin said in a statement.