Acceleron Receives FDA Orphan Drug Designation for ACE-083 in Charcot-Marie-Tooth Disease
“This formal designation aligns with our mission to develop therapies
for patients with rare diseases of high unmet medical need,” said
Orphan designation is granted by the
ACE-083 is currently being evaluated in two Phase 2 trials: one in CMT
and one in facioscapulohumeral muscular dystrophy (FSHD). Preliminary
results from Part 2 of the trials are expected by year end 2019 for CMT
and in the second half of 2019 for FSHD. During 2018, the
ACE-083, a locally-acting therapeutic candidate based on the naturally-occurring protein follistatin, utilizes the Myostatin+ approach to inhibit multiple TGF-beta superfamily ligands. It is designed to have a concentrated effect along targeted muscles to maximize growth and strength selectively in the muscles into which the drug is administered. Acceleron is developing ACE-083 for disorders such as CMT and FSHD, in which improved muscle strength in target muscles may provide a clinical benefit and enhance quality of life.
ACE-083 is an investigational therapy that is not approved for any use in any country.
CMT is one of the most common inherited neurologic diseases, estimated
to affect more than 125,000 people in the
Acceleron is a clinical-stage biopharmaceutical company dedicated to the discovery, development, and commercialization of therapeutics to treat serious and rare diseases. The Company's leadership in the understanding of TGF-beta biology and protein engineering generates innovative compounds that engage the body's ability to regulate cellular growth and repair.
Acceleron focuses its research and development efforts in hematologic, neuromuscular, and pulmonary diseases. In hematology, the Company and its global collaboration partner, Celgene, are developing luspatercept for the treatment of chronic anemia in myelodysplastic syndromes, beta-thalassemia, and myelofibrosis. Acceleron is also advancing its neuromuscular franchise with two distinct Myostatin+ agents, ACE-083 and ACE-2494, and a Phase 2 pulmonary program with sotatercept in pulmonary arterial hypertension.
This press release contains forward-looking statements about the Company's strategy, future plans and prospects, including statements regarding the development of the Company's compounds, the timeline for clinical development and regulatory approval of the Company’s compounds and the expected timing for reporting of data from ongoing clinical trials. The words "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "may," "plan," "potential," "project," "should," "target," "will," "would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.
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Acceleron Pharma Inc.
Todd James, IRC, 617-649-9393
Vice President, Investor Relations and Corporate Communications
Matt Fearer, 617-301-9557
Director, Corporate Communications