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Edgewise Therapeutics

Edgewise Therapeutics: Receives FDA Orphan Drug and Rare Pediatric Disease Designations

December 1, 2023

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

What is EDG-5506?

EDG-5506 is an investigational orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies, including individuals living with Duchenne and Becker. It seeks to protect the muscle from injuries due to hypercontractal stress when dystrophin is absent or has reduced function.

It 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. 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.”

Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise

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