BioMarin's Roctavian could change the landscape in severe hemophilia A, analyst says
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.
James Donagher, oncology and hematology analyst at GlobalData, said: “One-year results from the GENEr8-1 trial state that over 90% of the 134 patients experienced an annualized bleeding rate (ABR) of zero or lower than baseline after four weeks of treatment with Roctavian.
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