www.fdanews.com/articles/175640-biomarin-secures-orphan-drug-status-for-bmn-270-gene-therapy
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BioMarin Secures Orphan Drug Status for BMN 270 Gene Therapy
March 8, 2016
The FDA has awarded BioMarin orphan drug designation for its experimental gene therapy BMN 270 for the treatment of hemophilia A.
It is being studied in a five-year Phase 1/2 clinical trial in up to 12 patients with severe hemophilia A.