Acceleron DMD drug gets orphan drug status
Acceleron Pharma Inc. has won financial incentives and enhanced protection from generic competition, for its drug candidate to treat Duchenne Muscular Dystrophy (DMD), a fatal neuromuscular disease in which patients experience a progressive loss of muscle mass and strength.
The Cambridge-based biotechnology company says the U.S. Food and Drug Administration granted orphan status for the drug, called ACE-031, after determining it could meet an unmet medical need for patients of a rare disease.
The company has also received fast track status for the potential therapy, meaning that the FDA will shorten the time it takes to make a decision on approval, once the drug target is submitted. The drug target is currently being studied in a Phase 2 clinical trial for this indication.
Once a drug candidate has orphan status, the FDA may provide grant funding towards clinical trial costs, tax advantages, FDA user-fee benefits and seven years of market exclusivity in the U.S. following drug approval by the FDA.
DMD primarily affects boys and occurs in approximately 1 in every 3,500 live male births. Few patients survive beyond their late-20s when their heart and respiratory muscles weaken and eventually fail.