U.S. Food and Drug Administration Grants Orphan Drug Designation to Cytokinetics for CK-3773274 for the Treatment of Hypertrophic Cardiomyopathy
January 11, 2021 at 07:30 am EST
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Cytokinetics, Incorporated announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to CK-3773274 (CK-274) for the treatment of symptomatic hypertrophic cardiomyopathy (HCM). CK-274 is a next-generation cardiac myosin inhibitor in development for the potential treatment of HCM. 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 conditions 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. In preclinical models of cardiac function, CK-274 reduced cardiac contractility in a predictable dose and exposure dependent fashion. In preclinical models of disease, CK-274 reduced compensatory cardiac hypertrophy and cardiac fibrosis. The preclinical pharmacokinetics of CK-274 were characterized, evaluated and optimized for potential ease of titration in the clinical setting.
Cytokinetics, Incorporated is a late-stage, specialty cardiovascular biopharmaceutical company focused on discovering, developing and commercializing first-in-class muscle activators and next-in-class muscle inhibitors as potential treatments for debilitating diseases in which cardiac muscle performance is compromised. The Company specializes in muscle biology and the mechanics of muscle performance. It is developing small molecule drug candidates specifically engineered to impact myocardial muscle function and contractility. Its cardiac muscle contractility program is focused on the cardiac sarcomere. Its clinical-stage drug candidates are: aficamten, which is a next-in-class cardiac myosin inhibitor; omecamtiv mecarbil, which is a cardiac myosin activator; CK-586, which is an additional cardiac myosin inhibitor, and CK-136, which is a cardiac troponin activator. CK-586 is designed to reduce the hypercontractility associated with heart failure with preserved ejection fraction.