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DMD Gene Therapy Candidate RGX-202 Earns Orphan Drug Status

Posted on: 30 November 2021, source: Bionews
RGX-202, a one-time experimental gene therapy for the treatment of Duchenne muscular dystrophy (DMD), has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). The designation is given to investigational therapies to treat rare medical conditions or diseases that affect fewer than 200,000 people in the U.S. Orphan drug status provides benefits to support the process of developing new therapeutics, including tax credits for costs associated with clinical trials, exemptions from certain fees, and marketing exclusivity for seven years if the therapy achieves FDA approval.