PPMD is pleased to share that REGENXBIO Inc. announced this morning that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for RGX-202, a  gene therapy approach for the treatment of Duchenne muscular dystrophy (Duchenne). RGX-202 is designed to deliver a novel, optimized microdystrophin transgene with a unique C-terminal domain and a muscle specific promoter to support targeted therapy for improved resistance to muscle damage associated with Duchenne. RGX-202 uses REGENXBIO’s proprietary NAV® AAV8 vector. 

REGENXBIO expects to submit an Investigational New Drug (IND) application to the FDA for RGX-202 by the end of 2021. PPMD will be sure to share additional details on RGX-202 with the community as they become available.

FDA Orphan Drug Designation is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States. Orphan drug status provides  many benefits to drug developers and is an important achievement as this work progresses. 

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