Acceleron announced today that the US Food and Drug Administration (FDA) has granted Orphan Drug designation to ACE-083 for the treatment of patients with Charcot-Marie-Tooth disease (CMT). This designation gives Acceleron certain financial benefits that will help to lower the cost of developing the drug.
ACE-083 is currently being tested in a Phase 2 trial with results expected later this year. Positive preliminary data was announced in 2018.
For more information, please read Acceleron’s news release.