WASHINGTON (dpa-AFX) - BioMarin Pharmaceutical Inc. (BMRN) announced the U.S. FDA has granted Breakthrough Therapy Designation for valoctocogene roxaparvovec, an Investigational Gene Therapy for Hemophilia. BioMarin expects to initiate enrollment of a global Phase 3 program before the end of the year.
The Breakthrough Therapy Designation status was granted based on the data from an ongoing BioMarin Phase 1/2 study, which evaluated safety and efficacy of valoctocogene roxaparvovec.
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