Solid Biosciences Inc. (NASDAQ: SLDB) announced that the European Commission granted orphan drug designation to SGT-003 for treating Duchenne muscular dystrophy, according to a company statement.

The designation follows a positive opinion from the European Medicines Agency Committee for Orphan Medicinal Products. SGT-003 has previously received multiple regulatory designations including Fast Track, Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration, as well as Innovation License and Access Pathway designation in the U.K.

SGT-003 is currently being evaluated in two clinical trials: INSPIRE DUCHENNE, a Phase 1/2 trial, and IMPACT DUCHENNE, a Phase 3 randomized, double-blind, placebo-controlled trial. Both studies are designed to support potential regulatory authorizations in multiple countries.

The European Commission grants orphan drug designation for medicinal products treating life-threatening or chronically debilitating diseases affecting no more than five people in 10,000 in the EU. Designated drugs receive incentives including reduced regulatory fees, scientific assistance and 10 years of market exclusivity if approved.

Duchenne muscular dystrophy is a genetic muscle-wasting disease predominantly affecting boys, with symptoms typically appearing between ages three and five. The progressive disease affects approximately one in every 5,000 live male births and has an estimated prevalence of 10,000 to 15,000 cases in the United States.

SGT-003 is an investigational gene therapy containing a microdystrophin construct and a proprietary capsid designed to target integrin receptors. The therapy includes R16/17 domains intended to localize nNOS to muscle tissue.