1 May 2018 - Acceleron Pharma today announced that the U.S. 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.”

ACE-083 is currently being evaluated in two Phase 2 trials: one in FSHD and one in Charcot-Marie-Tooth (CMT) disease.

Read Acceleron Pharma press release