FDA Recognition for New Drug from Edgewise Therapeutics Marks Breakthrough for Duchenne and Becker Treatments
Edgewise’s investigational drug is a pioneering treatment for these muscular dystrophies.
Edgewise Therapeutics, a biopharmaceutical company specializing in muscle diseases, has achieved significant milestones in its muscular dystrophy program. The U.S. Food & Drug Administration (FDA) has granted Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) for the company's investigational drug, EDG-5506, designed to address Duchenne and Becker muscular dystrophies.
EDG-5506, an orally administered small molecule, aims to prevent contraction-induced muscle damage in dystrophinopathies. The FDA's ODD and RPDD designations emphasize the crucial need for improved therapeutic options for individuals grappling with these rare, serious, or life-threatening disorders.
Orphan Drug Designation (ODD) is granted to support the development of medicines for rare diseases affecting fewer than 200,000 people in the U.S. Potential benefits include market exclusivity for seven years upon FDA approval, federal tax credits, and waiver of Prescription Drug User Fee Act (PDUFA) fees. Rare Pediatric Disease Designation (RPDD) recognizes therapies under investigation for rare pediatric diseases affecting less than 200,000 people in the U.S., primarily impacting individuals up to 18 years of age. RPDD offers priority review of marketing applications and grants the sponsor a priority review voucher, transferable or sellable to another sponsor if approved for marketing.
Duchenne Muscular Dystrophy, a severe muscle disorder, and Becker Muscular Dystrophy, a progressive neuromuscular disorder, both lack effective cures. EDG-5506 presents a promising approach to minimize muscle damage, potentially benefiting patients suffering from these debilitating conditions.
Edgewise Therapeutics is actively advancing EDG-5506 through Phase 2 trials, known as LYNX and FOX, assessing safety, pharmacokinetics, and biomarkers of muscle damage in individuals with Duchenne. Additionally, the CANYON study, focusing on Becker, has been expanded to include a pivotal cohort called GRAND CANYON, currently enrolling participants. The company is committed to changing the lives of patients and families affected by serious muscle diseases. For more information on their clinical trials and mission, visit www.edgewisetx.com.
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