Acceleron receives FDA Orphan Drug Designation for ACE-083

Acceleron Pharm announced that the FDA has granted Orphan Drug designation to ACE-083, the company’s locally-acting “Myostatin+” muscle agent, for the treatment of patients with Charcot-Marie-Tooth disease. ACE-083 is currently being evaluated in two Phase 2 trials: one in CMT and one in facioscapulohumeral muscular dystrophy, or FSHD. Preliminary results from Part 2 of the trials are expected by year end 2019 for CMT and in the second half of 2019 for FSHD. During 2018, the FDA granted Fast Track designation for ACE-083 in CMT as well as Fast Track and Orphan Drug designations for ACE-083 for the treatment of patients with FSHD.
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