Acceleron announced the United States Food and Drug Administration (FDA) has granted orphan drug designation for ACE-083, the company’s locally acting “Myostatin+” muscle agent, for the treatment of patients with facioscapulohumeral muscular dystrophy (FSHD).
“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,” said Robert K. Zeldin, M.D., Chief Medical Officer of Acceleron. “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.”
Subscribe to our e-Newsletters
Stay up to date with the latest news, articles, and events. Plus, get special offers
from American Pharmaceutical Review – all delivered right to your inbox! Sign up now!
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 U.S. Under the Orphan Drug Act, the FDA may provide grant funding toward clinical trial costs, tax advantages, FDA user-fee benefits, and seven years of market exclusivity in the United States following marketing approval by the FDA. The granting of an orphan designation request does not alter the standard regulatory requirements and process for obtaining marketing approval.
In May of 2018, FDA granted ACE-083 Fast Track designation for FSHD, which could facilitate its development and potentially expedite its review. ACE-083 is currently being evaluated in two Phase 2 trials: one in FSHD and one in Charcot-Marie-Tooth (CMT) disease. The final Part 1 results from both Phase 2 trials are expected in the second of half of 2018. Preliminary results from Part 2 of the trial in patients with FSHD are expected in the second half of 2019.