Cytokinetics Receives FDA Orphan Drug Designation for CK-2127107 for SMA

Cytokinetics, Inc. recently announced that the Office of Orphan Products Development of the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to CK-2127107, a next-generation fast skeletal muscle activator, for the potential treatment of spinal muscular atrophy.

Orphan designation is granted to drugs and biologic products that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders. Through this designation, the FDA provides incentives for further clinical research and marketing authorization for conditions where there is a significant unmet need for effective therapies.

“We are pleased that the FDA has granted orphan drug status to CK-2127107 for the potential treatment of patients with SMA,” said Fady I. Malik, Cytokinetics’ Executive Vice President and Head of Research & Development.

CK-2127107 Clinical Trial Currently Recruiting

In March, Cytokinetics announced the opening of recruitment for a second cohort of their Phase 2 trial of CK-2127107. This Phase 2 double-blind, placebo-controlled clinical trial will enroll ambulatory and non-ambulatory patients 12 years of age and older, with SMA type II, III or IV. The trial will measure the drug’s effects on muscle strength and function, including respiratory assessments.

For additional information about the CK-2127101 clinical trial, visit

Cure SMA Funding for Combination Therapies

The clinical trials for CK-2127107 materialized because of early seed funding from Cure SMA supporting research focused on the potential application of these specific skeletal muscle activators. In 2014, Cytokinetics released encouraging data from preclinical studies conducted with our funding. The data showed this approach had positive effects in preserving muscle strength and reducing muscle fatigue, setting the groundwork for the ongoing clinical trials.

The progress of this program also highlights the importance of developing combination therapies to treat SMA. The goal is that CK-2127107 will show positive results in preserving muscle strength in human clinical trials, and may lend itself to combination with other SMA therapies, particularly those that address the SMN protein deficiency caused by the SMN1 mutation. The development of combination therapies is particularly important as we seek to treat all types, ages and stages of SMA.

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