Acceleron Pharma Inc. (NASDAQ:XLRN), a leading biopharmaceutical company in the discovery and development of TGF-beta therapeutics to treat serious and rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to ACE-083, a locally-acting “Myostatin+” muscle agent, for the treatment of patients with facioscapulohumeral muscular dystrophy (FSHD).
“This is an important milestone in the development of ACE-083—our lead program within our neuromuscular franchise,” said Matthew Sherman, Chief Medical Officer of Acceleron. “FSHD is a serious and rare neuromuscular disorder for which there are currently no approved therapies available. With this designation, we will be able to expedite the FDA review process of ACE-083, and if successful, deliver the first locally-acting, ‘Myostatin+’ muscle agent as a meaningful treatment option for the thousands of patients impacted by FSHD.”
The FDA’s Fast Track designation is a process designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier. Fast Track addresses a broad range of serious conditions. Once a drug receives Fast Track designation, early and frequent communication between the FDA and a drug company is encouraged throughout the entire drug development and review process. The frequency of communication assures that questions and issues are resolved quickly, often leading to earlier drug approval and access by patients.
ACE-083 is currently being evaluated in two Phase 2 trials: one in FSHD and one in Charcot-Marie-Tooth (CMT) disease.
Filed Under: Drug Discovery