Cytokinetics, a clinical-stage biopharm company developing novel small molecule therapeutics for the potential treatment of serious diseases and medical conditions, today announced that the European Medicines Agency (EMA) has granted the company’s lead drug candidate CK-2017357 orphan medicinal product designation for the treatment of amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig’s Disease.
CK-2017357 is currently in phase II clinical development program in patients with ALS, and has also been granted orphan drug designation by the U.S. Food and Drug Administration for the potential treatment of ALS, a debilitating disease of neuromuscular impairment.
Orphan drug designation offers several potential incentives, including the possibility of a 10-year period of EU marketing exclusivity from the date of marketing authorization, EU-funded research, protocol assistance, and fee reductions.
“We are pleased that the EMA has granted orphan medicinal product status to CK-2017357 for the treatment of ALS. This designation, along with a similar orphan drug designation already received in the U.S. from the U.S. 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,” Andrew A. Wolff, M.D, senior vice president of clinical research and development and chief medical officer of Cytokinetics stated in the press release. “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.”
The company reports that CK-2017357 demonstrated potentially clinically relevant pharmacodynamic effects in a phase IIa Evidence of Effect clinical trial in ALS patients, showing that the single doses of CK-2017357 evaluated were generally well-tolerated.
Cytokinetics has met with the FDA’s Center for Drug Evaluation and Research’s Division of Neurology Products, as well as with the EMA, to discuss its progress and future in the development of CK-2017357 as a potential treatment for patients with ALS, and said it anticipates conducting additional meetings with U.S. and European regulatory authorities during 2012 for further discussions.
For more information visit www.cytokinetics.com
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