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# Orphan Designation for Acceleron’s ACE-083
- URL: https://www.fdaweb.com/orphan-designation-for-accelerons-ace-083/
- Published: 2018-07-12T12:00:00.000Z
- Updated: 2026-09-15T00:19:02.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5141855

FDA has granted Acceleron Pharma an orphan drug designation for ACE-083, the company’s locally acting myostatin+ muscle agent, for treating patients with facioscapulohumeral muscular dystrophy (FSHD). The designation was based on positive preliminary data from an ongoing Phase 2 trial in patients with FSHD, according to the company. In May, the agency granted the therapy fast track status. ACE-083 is also currently being evaluated in Charcot-Marie-Tooth disease.