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FDA Grants Breakthrough Therapy Designation to Gene Therapy for Hemophilia A

December 30, 2021

The U.S. Food and Drug Administration granted breakthrough therapy designation to valoctocogene roxaparvovec (formerly BMN 270), an adeno-associated virus 5 factor VIII vector designed to restore factor VIII plasma concentrations in patients with hemophilia A, which is caused by a missing or deficient factor VIII.

The decision is supported by data from an ongoing study designed to evaluate the safety and efficacy of the investigational gene therapy. The drug's manufacturer expects to initiate enrollment of approximately 40 patients for its global phase III program this year. The program includes two studies of valoctocogene roxaparvovec at doses of 4e13 vg/kg and 6e13 vg/kg.

In March 2016, BMN 270 was granted orphan drug designation for hemophilia A.

Source: BioMarin Pharmaceutical press release, October 26, 2017.

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