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Regulatory Approval Sought for LDL Cholesterol-Lowering Drug Evolocumab

First BLA submission for PCSK9 inhibitor

A biologics license application (BLA) has been submitted to the FDA for evolocumab (Amgen), seeking approval 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. The drug is designed to bind to PCSK9 and to inhibit PCSK9 from binding to LDL receptors on the liver’s surface. In the absence of PCSK9, more LDL receptors are present on the surface of the liver to remove LDL-C from the blood.

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. These studies evaluated the safety and efficacy of evolocumab in patients with elevated cholesterol during treatment with statins with or without other lipid-lowering therapies; in patients who could not tolerate statins; in patients with heterozygous familial hypercholesterolemia (HeFH); and in patients with homozygous familial hypercholesterolemia (HoFH).

Elevated LDL-C is recognized as a major risk factor for cardiovascular disease. Familial hypercholesterolemia (FH) is an inherited condition caused by genetic mutations that lead to high levels of LDL-C at an early age, and it is estimated that less than 1% of people with FH in the U.S. are diagnosed.

Patients can have either one of two types of FH. HeFH is the more common type of FH and occurs in approximately one in 200 to 500 people. It can cause LDL-C levels two times higher than normal (e.g., > 190 mg/dL).Individuals with HeFH have one altered copy of a cholesterol-regulating gene. HoFH is a rare, more severe form of FH, occurring in approximately one in one million individuals. It can cause LDL-C levels more than six times higher than 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 the treatment of HoFH.

Source: Amgen; August 28, 2014.

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