New Therapy for Charcot-Marie-Tooth Disease Gets Orphan Drug Designation

ACE-083 May Improve Strength in Target Muscles, Enhance Quality of Life

The FDA has granted an orphan drug designation to Acceleron Pharma Inc.’s ACE-083, a locally acting “Myostatin+” muscle agent, for the treatment of Charcot-Marie-Tooth (CMT) disease.

Clinical trials to date have demonstrated that treatment with ACE-083 results in substantial increases in muscle volume in target muscles. If ongoing studies show that such increases lead to improved functional outcomes, ACE-083 could become an important new therapy for patients with CMT.

ACE-083 is being evaluated in two phase 2 trials: one in CMT and one in facioscapulohumeral muscular dystrophy (FSHD). Preliminary results from part 2 of the trials are expected by the end of 2019 for CMT and during the second half of 2019 for FSHD.

In 2018, the FDA granted fast-track designation for ACE-083 in CMT and fast-track and orphan drug designations for the treatment of patients with FSHD.

ACE-083 is a therapeutic candidate based on follistatin, which occurs naturally. The drug uses the Myostatin+ approach to obstruct multiple TGF-beta superfamily ligands, and is designed to have a concentrated effect to specifically maximize growth and strength in the muscles into which ACE-083 is administered.

CMT is one of the most common inherited neurologic diseases, estimated to affect more than 125,000 people in the United States. Symptoms include muscle weakness in the hands and lower legs, which can result in foot drop leading to frequent trips and falls. There are currently no approved therapies for CMT.

Source: Acceleron, March 5, 2019