Amgen submits BLA to FDA for novel cholesterol drug

Amgen has submitted the first Biologics Licence Application (BLA) to the US FDA for a PCSK9 inhibitor.

Its drug evolocumab is for the treatment of high cholesterol and 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) from the blood.

The drug is designed for hard-to-treat cases of raised cholesterol and is unlikely to take market share in the treatment of average patients with raised cholesterol, which is already well served by statins.

The BLA for evolocumab contains data from approximately 6,800 patients, including more than 4,500 patients with high cholesterol in 10 phase III trials. The 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), a rare and serious genetic disorder.

“This BLA submission to the FDA marks the first of several submissions to regulatory authorities around the world for our lipid-lowering programme and represents a critical milestone in our overall global development programme for evolocumab,” said Dr Sean Harper, executive vice president of Research and Development at Amgen.

High cholesterol, particularly elevated LDL-C, is the most common form of dyslipidemia, an abnormality of cholesterol and/or fats in the blood. Elevated LDL-C is 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.

Patients can have either one of two types of FH. Heterozygous FH is the more common type and occurs in approximately one in 200 to 500 people. It can cause LDL-C levels twice as high as normal 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. An individual with HoFH has two altered copies of cholesterol-regulating genes. In 2013, the FDA granted evolocumab an orphan drug designation for HoFH.

The clinical trial programme PROFICIO is evaluating evolocumab in 22 clinical trials, with a combined planned enrolment of approximately 30,000 patients.

Amgen’s cardiology pipeline includes several investigational molecules to address a number of unmet patient needs, such as high cholesterol and heart failure.

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Amgen cholesterol drug hits trial target

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