Acceleron Pharma Inc. (NASDAQ: XLRN) shares reached a multiyear high last month after the company and Celgene Corp. (NASDAQ: CELG) announced positive late-stage trial results, but those shares dipped on Friday after the company provided an update from the U.S. Food and Drug Administration (FDA).
Specifically, the FDA has granted an orphan drug designation for ACE-083, Acceleron’s locally acting “Myostatin+” muscle agent, for the treatment of patients with facioscapulohumeral muscular dystrophy (FSHD).
For some quick background: orphan designation is granted by the FDA Office of Orphan Products Development to advance the evaluation and development of safe and effective therapies for the treatment of rare diseases or conditions affecting fewer than 200,000 people in the United States.
Robert K. Zeldin, M.D., chief medical officer of Acceleron, commented:
We are pleased to receive orphan drug designation for ACE-083, which has shown the potential to address an area of high unmet medical need. We believe that ACE-083 could become an important new treatment for patients with FSHD whose muscle weakness negatively affects their functional abilities. We presented positive preliminary data from Part 1 of our Phase 2 trial in patients with FSHD earlier this year and look forward to sharing Part 2 results later next year.
Shares of Acceleron were last seen down about 2% at $46.15, with a consensus analyst price target of $57.00 and a 52-week range of $29.57 to $50.00.