FDA grants Edgewise ODD, RPDD for EDG-5506 to treat Duchenne, Becker MD

Edgewise Therapeutics announced that the U.S. Food & Drug Administration FDA has granted EDG-5506 Orphan Drug Designation ODD for the treatment of Duchenne muscular dystrophy or Duchenne, and Becker muscular dystrophy and Rare Pediatric Disease Designation or 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. “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.”