FDA Awards ARMGO Pharma’s ARM210 Orphan Drug and Rare Pediatric Disease Designations
The FDA has granted orphan drug and rare pediatric disease designations to ARMGO Pharma’s ARM210 for the treatment of Duchenne Muscular Dystrophy.
The oral small molecule targets the Ryanodine Receptor, an intracellular calcium release channel that becomes leaky, contributing to muscle damage and loss of function.
In Phase 1 trials, the therapy showed significant and robust improvements in exercise capacity, muscle specific force, grip strength and muscle histology. The treatment is part of a novel class of candidates called Rycals that ARMGO is developing to treat cardiac, musculoskeletal and neurological disorders.
An orphan drug designation is granted for the potential treatment of rare diseases or conditions affecting fewer than 200,000 patients. Under the FDA's rare pediatric disease priority review voucher program, a sponsor that receives an approval of an NDA for a rare pediatric disease may be eligible for a priority review voucher that can be redeemed for any subsequent marketing application. The voucher also can be sold or transferred.