August 19. 2010

Boston Business Journal

Acceleron advances MD drug


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, Mass.-based biotechnology company says the United States Food and Drug Administration 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 United States 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.