Edgewise Receives Orphan Drug and Rare Pediatric Disease Designations for Its Muscular Dystrophy Program from FDA

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[{"type":"text","content":" BOULDER, Colo.--(BUSINESS WIRE)--\nEdgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the U.S. Food & Drug Administration (FDA) has granted EDG-5506 Orphan Drug Designation (ODD) for the treatment of Duchenne muscular dystrophy (Duchenne) and Becker muscular dystrophy (Becker) and Rare Pediatric Disease Designation (RPDD) for the treatment of Duchenne. EDG-5506 is an investigational orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies, including Duchenne and Becker. EDG-5506 is currently advancing in multiple Phase 2 trials for individuals with Duchenne, Becker and other dystrophinopathies. The FDA previously granted Fast Track designation for the investigation and development of EDG-5506 for the treatment of Becker.\n\n\n“Receiving orphan drug and rare pediatric disease designations are important milestones in advancing our novel small molecule therapeutic approach to treating individuals with Duchenne and Becker,” said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise. “These regulatory designations highlight the urgent and critical need for new and better therapeutic options for people living with these rare, serious or life-threatening disorders.”\n\n\nBenefits of ODD and RPDD\n\n\nThe FDA grants ODD to support development of medicines for rare diseases or conditions that affect fewer than 200,000 people in the U.S. Potential benefits of the ODD include market exclusivity for the first ODD drug for an approved indication within the ODD for a seven-year period upon FDA approval, federal tax credit for qualified clinical research expenses incurred in the U.S., and a waiver of Prescription Drug User Fee Act (PDUFA) fees (currently worth over $4 million).\n\n\nRPDD acknowledges therapies under investigation for rare pediatric diseases affecting less than 200,000 people in the U.S. with serious or life-threatening manifestations primarily affecting individuals up to 18 years of age. RPDD provides priority review of the marketing application, and, if approved for marketing, grants that sponsor a priority review voucher which can be transferred or sold to another sponsor.\n\n\nAbout Duchenne Muscular Dystrophy\n\n\nDuchenne is a severe, degenerative muscle disorder with a median life expec...

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