Acceleron Receives FDA Fast Track Designation for ACE-083 in Charcot-Marie-Tooth Disease
Acceleron Pharma Inc. (NASDAQ:XLRN), a leading biopharmaceutical company in the discovery and development of TGF-beta therapeutics to treat serious and rare diseases, today announced that the United States Food and Drug Administration (FDA) has granted Fast Track designation to ACE-083, the Company’s locally-acting “Myostatin+” muscle agent, for the treatment of patients with Charcot-Marie-Tooth disease (CMT).
“We’re pleased that the FDA has granted this designation for ACE-083,” said Robert K. Zeldin, M.D., Chief Medical Officer of Acceleron. “Patients with CMT currently have no approved therapies. To date, results from our Phase 2 trials have shown that patients treated with ACE-083 experience robust increases in muscle volume. If our ongoing clinical studies show that ACE-083 also improves functional outcomes and confirm the favorable safety profile observed thus far, the Fast Track process could help us work with the FDA to deliver it to patients as quickly as possible.”
The FDA’s Fast Track designation is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. Once a drug receives Fast Track designation, early and frequent communication between the FDA and a drug company is encouraged throughout the entire drug development and review process. The frequency of communication assures that questions and issues are resolved quickly, often leading to earlier drug approval and access by patients.
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. Earlier this year, the FDA granted Fast Track and Orphan Drug designations for ACE-083 for the treatment of patients with FSHD.
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.
CMT is the most common inherited neurologic disease, estimated to affect more than 125,000 people in the United States. The primary clinical manifestations of CMT include muscle weakness in the lower legs and hands. The lower leg muscle weakness can result in foot drop leading to frequent trips and falls. The disease is typically diagnosed by a characteristic pattern of muscle weakness and sensory deficits, family history, nerve conduction studies, and genetic testing. There are no FDA-approved therapies for CMT.
Acceleron is a Cambridge-based, 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.
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