BioMarin's Roctavian could change the landscape in severe hemophilia A, analyst says

9 August 2021

US biotech BioMarin Pharmaceutical (Nasdaq: BMRN) recently presented positive one-year results from its GENEr8-1 Phase III trial, in which adults with severe hemophilia A are treated with the gene therapy Roctavian (valoctocogene roxaparvovec).

If approved, patients with severe hemophilia A will have a chance at a normal life with no, or at least very few, ongoing medical interventions, albeit having to pay a high list price, says the data and analytics company GlobalData.

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