Cytokinetics, Incorporated announced that its fast skeletal muscle troponin activator, CK-2017357, has been granted orphan-drug designation by the US Food and Drug Administration (FDA) for the potential treatment of amyotrophic lateral sclerosis (ALS), also commonly known as Lou Gehrig's Disease. CK-2017357 is the lead drug candidate that has emerged from the company's skeletal sarcomere activator program. Cytokinetics plans to initiate a phase-II Evidence of Effect clinical trial for CK-2017357 in ALS patients in the first half of 2010.
Orphan-drug designation is granted by the US FDA Office of Orphan Drug Products Development to novel drugs or biologics that may treat a condition affecting less than 200,000 persons in the United States or occurs in more than 200,000 persons and for which there is no reasonable expectation that the cost of development and distribution of the drug will be recovered. The designation offers a number of potential incentives, which may include a seven-year period of US marketing exclusivity from the date of marketing authorization, funding for clinical studies, study design assistance, waiver of US FDA user fees, and tax credits for clinical research.
"We are pleased that the US FDA has granted orphan drug status to CK-2017357 for the potential treatment of ALS. This designation indicates their recognition that this novel drug candidate may address significant unmet medical needs in patients suffering from this grievous and uniformly fatal disease," said Andrew A Wolff, senior vice president and chief medical officer of Cytokinetics. "With the planned initiation of our Phase II Evidence of Effect trial in patients with ALS, we look forward to continuing to work closely with regulators, as well as with our clinical investigators and key opinion leaders in the field of ALS, to advance this promising drug candidate through clinical development."
Cytokinetics is a clinical-stage biopharmaceutical company focused on the discovery and development of small molecule therapeutics that modulate muscle function for the potential treatment of serious diseases and medical conditions.