Edgewise Therapeutics, Inc. announced that the U.S. Food & Drug Administration has granted EDG-5506 Orphan Drug Designation 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. The 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). RPDD 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. 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.

Becker is a genetic, progressive neuromuscular disorder that imposes significant physical, emotional, financial, and social impacts predominantly on males and their caregivers. Genetic mutations in the dystrophin gene resulting in Becker lead to contraction-induced muscle damage, which is the primary driver of muscle loss and impaired motor function in muscular dystrophies. Functional decline can begin at any age, and once that muscle loss occurs, the decline in function is irreversible and continues throughout the individual?s life.

Some individuals living with Becker experience heart failure from cardiomyopathy, which may result in heart transplantation or early death. Currently, there is no cure for Becker; early and long-term multidisciplinary care from neuromuscular specialists, cardiologists, physical therapists, and other specialists is critical for optimized disease management. Novel therapies are in development for Becker, including muscle targeted interventions, aimed at positively impacting disease trajectory.