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FDA Grants Orphan Status for Neuromuscular Drug

Acceleron Pharma, Inc., a biopharmaceutical company in Cambridge, Massachusetts, today said the U.S. Food and Drug Administration (FDA) granted ACE-031, its treatment for Duchenne Muscular Dystrophy (DMD), with an orphan drug designation.

DMD is a debilitating and fatal genetic disorder characterized by the progressive loss of muscle strength and function. The disease affects mainly boys and occurs in approximately 1 in every 3,500 live male births. Acceleron Pharma says 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. ACE-031 is currently being studied in a phase 2 clinical trial of patients with DMD.

The FDA designates drugs as orphan drugs to 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, user-fee benefits, and seven years of market exclusivity in the United States following drug approval by the FDA.

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