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FDA grants breakthrough therapy designation to valoctocogene roxaparvovec for hemophilia A

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The FDA granted breakthrough therapy designation to valoctocogene roxaparvovec for the treatment of patients with hemophilia A.

The agency’s decision included data from an ongoing phase 1/phase 2 study, designed to evaluate the safety and efficacy of valoctocogene roxaparvovec (BMN 270, BioMarin Pharmaceutical) an investigational gene therapy.

BioMarin expects to initiate enrollment of approximately 40 patients for its global phase 3 program this year. The program includes two studies of valoctocogene roxaparvovec at doses of 4e13 vg/kg and 6e13 vg/kg.

“There is a tremendous need to achieve normal steady state factor VIII levels to eliminate spontaneous bleeding, to avoid the complications of suboptimally corrected bleeding disorder, to improve quality of life and enable patients to live to their fullest potential,” Hank Fuchs, MD, president of worldwide research and development at BioMarin, said in a company-issued release.