Acceleron Pharma, Inc., a biopharmaceutical company developing novel therapeutics that modulate the growth of cells and tissues including muscle, bone, fat, red blood cells and the vasculature, today announced the FDA granted orphan designation for ACE-031 for the treatment of Duchenne Muscular Dystrophy (DMD), a fatal neuromuscular disease in which patients experience a progressive loss of muscle mass and strength. ACE-031 is an investigational protein therapeutic being developed to increase muscle mass and strength.
“In the past two weeks, the FDA has granted both orphan status and Fast Track designation to ACE-031 in recognition of the drug’s potential to address the enormous unmet medical need for therapeutics to treat DMD patients”, said Matthew Sherman, M.D., Chief Medical Officer at Acceleron. “We will continue to work collaboratively with clinical investigators, health authorities and patient advocacy groups around the world to develop ACE-031.” Orphan drug designation is granted by the FDA Office of Orphan Products Development to assist and encourage companies to develop safe and effective therapies for the treatment of rare diseases and disorders. Under the Orphan Drug Act, 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. The approval of an orphan designation request does not alter the standard regulatory requirements and process for obtaining marketing approval. For more information about orphan designation, please visit the FDA website at www.fda.gov.