Amgen (NASDAQ: AMGN) today announced that the Company will discuss the data supporting the RepathaTM (evolocumab) Biologics License Application (BLA) for the treatment of high cholesterol with the U.S. Food and Drug Administration's (FDA) Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC). Repatha is an investigational fully human monoclonal antibody that inhibits proprotein convertase subtilisin/kexin type 9 (PCSK9), a protein that reduces the liver's ability to remove low-density lipoprotein cholesterol (LDL-C), or "bad" cholesterol, from the blood.
At today's FDA advisory committee meeting, Amgen will present Repatha clinical trial data from approximately 6,800 patients, including more than 4,500 patients with high cholesterol in 10 Phase 3 trials. The Phase 3 studies evaluated the efficacy and safety of Repatha in patients with elevated cholesterol, including patients on statins with or without other lipid-lowering therapies; patients who cannot tolerate statins; patients with heterozygous familial hypercholesterolemia (HeFH) and patients with homozygous familial hypercholesterolemia (HoFH), a rare and serious genetic disorder.
"We look forward to discussing the efficacy and safety data from our clinical program with the FDA advisory committee as there remains a significant unmet medical need for patients who, despite currently available therapies, are unable to control their high cholesterol," said Sean E. Harper, M.D., executive vice president of Research and Development at Amgen. "If approved, Repatha would provide patients and physicians with an important new treatment option for managing high cholesterol."
The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of Aug. 27, 2015, for the Repatha BLA.
High cholesterol, particularly elevated LDL-C, is the most common form of dyslipidemia, which is an abnormality of cholesterol and/or fats in the blood. Nearly one in three Americans have elevated LDL-C, which is recognized as a major risk factor for cardiovascular disease. Familial hypercholesterolemia (FH) is an inherited condition caused by genetic mutations which lead to high levels of LDL-C at an early age, and it is estimated that less than one percent of people with FH (heterozygous and homozygous forms) in the U.S. are diagnosed.
Liver receptor mutations linked to liver dysfunction, cholesterol accumulation, and hepatitis