CK-2017357, the lead drug candidate that has emerged from the Cytokinetics’ skeletal sarcomere activator program, is currently the subject of an ongoing Phase II clinical development program in patients with ALS.
The EMA approval for the designation offers a number of potential incentives, which may include a ten-year period of EU marketing exclusivity from the date of marketing authorization, EU-funded research, protocol assistance and fee reductions.
Cytokinetics senior vice president of Clinical Research and Development and chief medical officer Andrew Wolff said this designation, along with a similar orphan drug designation already received in the US from the US Food and Drug Administration, underscores the potential for this novel drug candidate to address significant unmet medical needs in patients suffering from this grievous and uniformly fatal disease.
"We look forward to working closely with the relevant regulatory authorities, as well as with our clinical investigators and key opinion leaders in the field of ALS, to advance this important and promising drug candidate rapidly through the next stages of clinical research and development," Wolff said.
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease with average life expectancy of an ALS patient is approximately three to five years and only 10% of patients survive for more than 10 years.
Respiratory failure because of diminished strength in the skeletal muscles responsible for breathing is the primary cause for death in this disease.
Under the American Recovery and Reinvestment Act of 2009, Cytokinetics was awarded a grant of approximately $2.8m in July 2010 from the National Institute of Neurological Disorders and Stroke to support research and development of CK-2017357 in myasthenia gravis.