Cytokinetics Granted Orphan Drug Designation for Reldesemtiv for the Treatment of Amyotrophic Lateral Sclerosis

SOUTH SAN FRANCISCO, Calif., Dec. 18, 2019 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq: CYTK) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to reldesemtiv for the treatment of amyotrophic lateral sclerosis (ALS). Previously reldesemtiv was granted orphan drug designation for the treatment of spinal muscular atrophy (SMA) by the FDA and by the European Medicines Agency. In collaboration with Astellas, Cytokinetics is developing reldesemtiv, a fast skeletal muscle troponin activator (FSTA), as a potential treatment for people with ALS, SMA and certain other debilitating diseases and conditions associated with skeletal muscle weakness and/or fatigue.

The FDA, through its Office of Orphan Products Development (OOPD), grants orphan status to drugs and biologic products that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. Orphan drug designation provides a drug developer with certain benefits and incentives, including a seven-year period of U.S. marketing exclusivity from the date of marketing authorization, waiver of FDA user fees, and tax credits for clinical research.

“We’re pleased that reldesemtiv received orphan designation from the FDA,” said Fady I. Malik, M.D., Ph.D., Cytokinetics’ Executive Vice President of Research & Development. “It’s an exciting time for the development of investigational medicines for ALS. We believe treatment with reldesemtiv may represent a complementary approach to other potential therapies by directly addressing impaired muscle function and weakness that affects patients with ALS.”  

About Reldesemtiv

Skeletal muscle contractility is driven by the sarcomere, the fundamental unit of skeletal muscle contraction and a highly ordered cytoskeletal structure composed of several key proteins. Skeletal muscle myosin is the motor protein that converts chemical energy into mechanical force through its interaction with actin. A set of regulatory proteins, which includes tropomyosin and several types of troponin, make the actin-myosin interaction dependent on changes in intracellular calcium levels. Reldesemtiv, a next-generation FSTA arising from Cytokinetics’ skeletal muscle contractility program, slows the rate of calcium release from the regulatory troponin complex of fast skeletal muscle fibers, which sensitizes the sarcomere to calcium, leading to an increase in skeletal muscle contractility. Reldesemtiv has demonstrated pharmacological activity that may lead to new therapeutic options for diseases associated with skeletal muscle weakness and fatigue.