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.

"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.

About ACE-031

ACE-031 is an investigational protein therapeutic that builds muscle and increases strength by inhibiting signaling through a cell surface receptor called activin receptor type IIB (ActRIIB). ACE-031 is a recombinant fusion protein that is produced by joining a portion of the human ActRIIB receptor to a portion of a human antibody. This creates a freely circulating, decoy version of ActRIIB which removes proteins, such as GDF-8 (myostatin), that limit the growth and regeneration of muscle. Animal studies with ACE-031 suggest that blocking signaling through ActRIIB leads to increased muscle mass and improved physical function in a range of animal models of muscle disease, including models of muscular dystrophy. ACE-031 is currently being studied in a phase 2 clinical trial of patients with Duchenne Muscular Dystrophy (DMD) who are also receiving corticosteroid therapy.

About Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy (DMD) is a debilitating and fatal genetic disorder characterized by the progressive loss of muscle strength and function. It primarily affects boys and occurs in approximately 1 in every 3,500 live male births. DMD is caused by genetic mutations that result in the absence or a defect in dystrophin, a protein necessary to maintain the structural integrity of muscle fibers. This condition leads to deterioration of and damage to skeletal muscles, which eventually become infiltrated by non-functional scar and fatty tissue. As a result, patients experience a relentless decline in muscle strength that impairs their ability to walk, breathe and live independently. Many patients spend the majority of their lives confined to wheelchairs and eventually lose all upper body function. Few patients survive beyond their late-20s when their heart and respiratory muscles weaken and eventually fail.

Source:
Acceleron Pharma, Inc.