Acceleron Pharma announced that the Food and Drug Administration (FDA) has granted Fast Track designation to ACE-083 for the treatment of patients with facioscapulohumeral muscular dystrophy (FSHD). 

FSHD is a rare genetic muscle disorder that is characterized by debilitating skeletal muscle weakness and loss. There is currently no approved therapies to treat FSHD. ACE-083 is a locally-acting “Myostatin+” muscle agent currently under development for FSHD. The therapeutic candidate is derived from the naturally-occurring protein follistatin and uses the “Myostatin+” approach to inhibit multiple TGF-beta ligands. Its concentrated effect along targeted muscles helps maximize growth and strength for the specific muscles in which the drug is delivered. 

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The FDA’s Fast Track designation helps facilitate drug development and expedite the review of drugs for serious conditions and unmet medical needs. If approved, ACE-083 will be the first locally-acting “Myostatin+” muscle agent for patients with FSHD.

ACE-083 is currently being studied in a Phase 2 trial for FSHD and another for Charcot-Marie-Tooth (CMT) disease. 

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