Facioscapulohumeral Muscular Dystrophy Treatment Gets Fast Track Status
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.
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.
For more information call (617) 649-9200 or visit AcceleronPharma.com.