Edgewise Therapeutics, Inc. 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 Dukehenne and Becker, Rare Pediatric Disease Designation (RPDD) for the treatment ofuchenne, and Fast Track designation for the treatment of Becker.

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 function, 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 is advancing EDG-5506 through the clinic including completing enrollment of a Phase 2 trial cohort, called CANYON, evaluating safety and effects on function and biomarkers of muscle damage in adult males with Becker, which has been expanded to include an additional 120 adult participants in a pivotal cohort called GRAND CANYON, which is currently enrolling. In Duchenne, the Company is advancing its Phase 2 clinical trials, LYNX, assessing safety, pharmacokinetics and biomarkers of muscle damage, and FOX, which includes children and adolescents previously treated with gene therapy.

For more information on Edgewise's clinical trials.