FDA grants Acceleron's ACE-031 orphan designation for treatment of DMD

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 United States Food and Drug Administration (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.

“We will continue to work collaboratively with clinical investigators, health authorities and patient advocacy groups around the world to develop ACE-031.”

"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."