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 it received FDA Fast Track designation for ACE-031 for the treatment of Duchenne Muscular Dystrophy (DMD), a fatal neuromuscular disease in which patients suffer from the progressive loss of muscle mass and strength. ACE-031 is an investigational protein therapeutic being developed to increase muscle mass and strength.

The Fast Track program of the FDA is designed to facilitate the development and expedite the review of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs. Fast Track designated drugs typically qualify for priority review which can further expedite the FDA review process.

“Acceleron is working diligently to develop ACE-031 for patients suffering from DMD,” said Matthew Sherman, M.D., Chief Medical Officer at Acceleron. “We are pleased the FDA recognizes the potential of ACE-031 to address the enormous unmet medical need for therapeutics to treat DMD patients. We will continue to work collaboratively with clinical investigators, health authorities and patient advocacy groups around the world to develop ACE-031.”

About the Phase 2 Clinical Trial in Duchenne Muscular Dystrophy

The Phase 2 trial is a randomized, double-blind, placebo-controlled, dose-ranging study to evaluate the safety, tolerability and pharmacokinetics of ACE-031 in patients with DMD receiving concurrent corticosteroid treatment. In addition, the study will characterize the effects of ACE-031 on lean tissue (muscle) mass, muscle strength, muscle function, pulmonary function and quality of life. In a single-dose Phase 1 study of ACE-031 in healthy post-menopausal women, rapid and sustained dose-dependent increases in lean mass and muscle volume were observed.

The study is being conducted at multiple sites in Canada. For a more detailed description of the clinical trial protocol, inclusion and exclusion criteria, and a list of participating sites, please visit and query NCT01099761 as the study identifier.