CMT drug receives Orphan Drug designation

Published Date
Beatriz Bustillo Ramirez
Open orange bottle containing white pills

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.

If you have any questions about this news story or any other CMT research, please contact the MDUK Research Line on 020 7803 4813 or email

Keep in touch