ARMGO Pharma announced the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to ARM210 (also known as S48168), a potential treatment for patients with Ryanodine Receptor Type 1 Related Myopathies (RYR1-RM). In 2015, the FDA granted orphan drug designation and rare pediatric disease designation to ARM210 as a potential treatment for Duchenne Muscular Dystrophy (DMD).

ARM210 targets the Ryanodine Receptor calcium-release channel (RyR), an intracellular calcium release channel that becomes leaky in a number of diseases including DMD and RYR1-RM, contributing to muscle damage and loss of function. RYR1-RM comprise a group of rare skeletal muscle diseases due to mutations in the ryr1 gene, which lead to leaky channels. These leaks both impair muscle contraction leading to muscle weakness and loss of function, and activate toxic pathways that damage muscle, causing the symptoms in RYR1-RM.

ARM210 is a small molecule that binds to leaky RyR channels and repairs the leak, as demonstrated in vitro in muscle biopsies from RYR1-RM patients. The unique mechanism of action of ARM210 makes it an ideal potential therapy for RYR1-RM.

"This designation represents an important achievement for the Rycal ARM210 program and highlights the unmet need to provide effective treatment for patients with RYR1-RM," said Gene Marcantonio MD PhD, President and Chief Medical Officer of ARMGO Pharma. "We are looking forward to continuing the progress of the ARM210 clinical program, including upcoming studies in RYR1-RM patients."

To apply for becoming a contributor of En-CPhI.cn,

welcome to send your CV and sample works to us,

Email: Julia.Zhang@ubmsinoexpo.com.