FDA accepts Amgen's evolocumab BLA for review

Amgen (NASDAQ: AMGN) today announced that the U.S. Food and Drug Administration (FDA) has accepted for review Amgen's Biologics License Application (BLA) for evolocumab for the treatment of high cholesterol. Evolocumab 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.

"There is still a large unmet need among patients with high cardiovascular risk and elevated cholesterol who are unable to reach optimal LDL cholesterol levels with current therapies," said Sean E. Harper, M.D., executive vice president of Research and Development at Amgen. "Evolocumab has the potential to provide significant additional benefit when added to existing LDL cholesterol-lowering medications for patients with high cholesterol."

The BLA, submitted on Aug. 27, 2014, is based on 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 safety and efficacy of evolocumab 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).

The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of Aug. 27, 2015, for the evolocumab application.

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. Elevated LDL-C 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.

Patients can have either one of two types of FH. Heterozygous FH is the more common type of FH and occurs globally in approximately one in 200 to 500 people. 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., 650-1,000 mg/dL). An individual with HoFH has two altered copies of cholesterol-regulating genes (one from each parent). In 2013, the FDA granted evolocumab an orphan drug designation for HoFH.