Acceleron Receives FDA Fast Track Designation for ACE-083 in Facioscapulohumeral Muscular Dystrophy (FSHD)

“This is an important milestone in the development of ACE-083—our lead program within our neuromuscular franchise,” said Matthew Sherman, Chief Medical Officer of Acceleron. “FSHD is a serious and rare neuromuscular disorder for which there are currently no approved therapies available. With this designation, we will be able to expedite the FDA review process of ACE-083, and if successful, deliver the first locally-acting, ‘Myostatin+’ muscle agent as a meaningful treatment option for the thousands of patients impacted by FSHD.”

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. The purpose is to get important new drugs to the patient earlier. Fast Track addresses a broad range of serious conditions. 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 FSHD and one in Charcot-Marie-Tooth (CMT) disease.

About ACE-083

ACE-083 is a locally-acting therapeutic candidate, based on the naturally-occurring protein follistatin, which utilizes the “Myostatin+" approach to inhibit multiple TGF-beta 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 diseases such as facioscapulohumeral muscular dystrophy (FSHD) and Charcot-Marie-Tooth (CMT) disease, in which improved muscle strength in target muscles may provide a clinical benefit and enhance quality of life.

About Facioscapulohumeral Muscular Dystrophy (FSHD)

FSHD is a rare genetic muscle disorder affecting approximately 20,000 people in the United States for which there are currently no approved treatments. The primary clinical presentation of FSHD is debilitating skeletal muscle weakness and loss. The symptoms of FSHD develop in a descending pattern, beginning with the face and upper body and progressing to the lower body in a “muscle by muscle” fashion. The disease is typically diagnosed by a characteristic pattern of muscle weakness and other clinical symptoms, as well as through genetic testing.

About Acceleron

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 pulmonary program with a Phase 2 trial of sotatercept planned in pulmonary arterial hypertension.

For more information, please visit www.acceleronpharma.com. Follow Acceleron on Social Media: @AcceleronPharma and LinkedIn.

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Source: Acceleron Pharma

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