Amgen (NASDAQ: AMGN) today announced that an application seeking marketing approval of RepathaTM (evolocumab) for the treatment of high cholesterol has been submitted for review to the Ministry of Health, Labour and Welfare in Japan. Repatha is being developed in Japan by Amgen Astellas BioPharma K.K., a joint venture between Amgen and Astellas Pharma Inc., a pharmaceutical company headquartered in Tokyo.
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. In Japan, LDL-C levels are not adequately controlled for many patients taking statins, nearly half of whom have not reached their LDL-C goal.
"Submitting Repatha for marketing approval in Japan is an important milestone in our strategic partnership alliance with Astellas Pharma as we look forward to accomplishing our common goal of addressing the critical needs of patients with high cholesterol," said Sean E. Harper, M.D., executive vice president of Research and Development at Amgen. "We look forward to working with regulatory authorities in Japan to provide a new treatment option for patients whose cholesterol is uncontrolled with currently available therapies."
The Japanese New Drug Application for marketing approval for Repatha contains data from approximately 7,200 patients with high cholesterol in 11 Phase 3 trials, including Japanese patients from studies conducted in Japan. Overall, the Phase 3 studies evaluated the safety and efficacy of Repatha in patients with elevated cholesterol 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.
In the U.S., Amgen submitted a Biologics License Application for Repatha for the treatment of high cholesterol to the Food and Drug Administration (FDA) in August 2014. The FDA's Prescription Drug User Fee Act target action date is Aug. 27, 2015. In the European Union, Amgen submitted a Marketing Authorization Application to the European Medicines Agency via the centralized procedure for Repatha for the treatment of high cholesterol in September 2014.
High cholesterol is the most common form of dyslipidemia, which is an abnormality of cholesterol and/or fats in the blood. There are approximately 300 million cases of dyslipidemia in the U.S., Japan and Western Europe.
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 Japan are diagnosed. Patients can have either one of two types of FH. Heterozygous FH is the more common type of FH and in Japan, occurs in approximately one in 900 individuals. It can cause LDL-C levels twice as high as normal (e.g., >190 mg/dL). Individuals with HeFH have one altered copy of a cholesterol-regulating gene. Homozygous FH is the rare, more severe form, occurring in approximately one in a million individuals. It can cause LDL-C levels more than six times as high as normal (e.g., 500-1,000 mg/dL). An individual with HoFH has two altered copies of cholesterol-regulating genes (one from each parent).
Study points to the possibility of using cholesterol-lowering drugs to treat aggressive colorectal tumors