Edgewise Receives U.S. FDA Fast Track Designation for EDG-5506 for the Treatment of Duchenne Muscular Dystrophy (Duchenne)

Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for EDG-5506 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 muscular dystrophy (Becker). The FDA previously granted EDG-5506 Orphan Drug Designation (ODD) for the treatment of Duchenne and Becker, Rare Pediatric Disease Designation (RPDD) for the treatment of Duchenne, and Fast Track designation for the treatment of Becker.

“Every day is important for individuals living with Duchenne and their families, and we are pleased that the FDA granted Fast Track designation,” said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise. “Our roster of regulatory designations granted to EDG-5506 highlights the urgent and critical need for new and better therapeutic options for people living with these rare and life-threatening muscle disorders.”

The FDA Fast Track Program is designed to facilitate the development and expedite the review of new therapeutics that are intended to treat serious conditions and fill an unmet medical need. The designation is granted to qualifying therapeutics based on factors such as potential to offer meaningful impact on survival, day-to-day functioning, or the likelihood that the condition, if left untreated, will progress to a more serious condition. Therapeutics that receive this designation receive important benefits that include more frequent meetings with the FDA to discuss development of the drug candidate and rolling review of the marketing application for obtaining FDA approval.

Ongoing trials in Duchenne include the Phase 2 placebo-controlled LYNX trial in children aged 4 to 9 years with Duchenne (NCT05540860), and the Phase 2 placebo-controlled FOX trial in children and adolescents with Duchenne who have been previously treated with gene therapy (NCT06100887). The Company expects to report LYNX 3-month controlled dose-ranging data in the second quarter of 2024, once the Phase 3 dose is identified.

About Duchenne Muscular Dystrophy

Duchenne is a severe, degenerative muscle disorder with a median life expectancy of around 30 years old. People living with Duchenne begin to lose their ability to walk without assistance by their early teens and nearly all will require the use of a wheelchair by the time they are in their mid-teens. Duchenne is the most common type of muscular dystrophy, and genetic mutations in the dystrophin gene result in contraction-induced muscle damage, which is the primary driver of irreversible muscle loss and impaired motor function. Currently, there is no cure for Duchenne; early, active multidisciplinary care from neuromuscular specialists, cardiologists, physical therapists, and other specialists is critical for optimized disease management. Current therapeutic options for Duchenne are inadequate to prevent significant morbidity and mortality; novel therapies in development for Duchenne, including muscle targeted interventions, aim to positively impact disease trajectory.