Regenxbio Gene Therapy Candidate Gets Fast Track Status for Duchenne Muscular Dystrophy

The FDA has granted Fast Track designation to Regenxbio’s RGX-202 candidate as a potential one-time gene therapy treatment for Duchenne muscular dystrophy.

The disorder, which is characterized by progressive muscular weakness, is caused by a genetic mutation that affects the production of dystrophin, resulting in cell damage during muscle contraction. RGX-202 introduces a novel gene to make muscles resistant to the contraction caused by the lack of dystrophin.

The gene therapy candidate is being evaluated in a phase 1/2 clinical trial, in which six patients ages four to 11 years are expected to enroll for a one-time intravenous dose of the medication.

The FDA grants the Fast Track designation to facilitate development and expedite review of new therapeutics to treat serious or life-threatening conditions and to address unmet medical needs.

The agency previously granted RGX-202 Orphan Drug and Rare Pediatric Disease designations.

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