Amgen Inc. of thousand Oaks, California has been given FDA approval for Repatha (evolocumab) injection for some patients who are unable to get their low-density lipoprotein (LDL) cholesterol under control with current treatment options. It is the 2nd drug in a new class of drugs known as PCSK9 inhibitors, to be granted governmental endorsement for use in addition to diet and maximally-tolerated statin therapy in adult patients with heterozygous familial hypercholesterolemia (HeFH), homozygous familial hypercholesterolemia (HoFH), or clinical atherosclerotic cardiovascular disease, such as heart attacks or strokes, who require additional lowering of LDL cholesterol.
Familial hypercholesterolemia (encompassing both HeFH and HoFH) is an inherited condition that causes high levels of LDL cholesterol. A high level of LDL cholesterol in the blood is linked to cardiovascular or heart disease. Heart disease is the number 1 cause of death for both men and women in the United States. According to the CDC nearly 610,000 people die of heart disease here annually, equal to 1 in every 4 deaths.
Repatha is described as an antibody that targets a specific protein, called PCSK9. PCSK9 reduces the number of receptors on the liver that remove LDL cholesterol from the blood.
By blocking PCSK9’s ability to work, more receptors are available to get rid of LDL cholesterol from the blood,” explained Dr. John Jenkins, M.D., director of the Office of New Drugs, Center for Drug Evaluation and Research. “Cardiovascular disease is a serious threat to the health of Americans, and the FDA is committed to facilitating the development and approval of effective and safe drugs to address this important public health problem.”
The efficacy and safety of Repatha were evaluated in a single placebo-controlled trial for 1-year, as well as 8 4-month placebo-controlled trials in participants with primary hyperlipidemia, including two that specifically enrolled participants with HeFH and one that enrolled participants with HoFH. In 1 of the 12-week studies, 329 participants with HeFH, who required additional lowering of LDL cholesterol despite statins with or without other lipid-lowering therapies, were randomized to receive Repatha or placebo for 12 weeks. Participants taking Repatha had an average reduction in LDL cholesterol of approximately 60%, compared to placebo. In addition, several clinical trials have demonstrated that statins lower the risk of having a heart attack or stroke. A trial evaluating the effect of adding Repatha to statins for reducing cardiovascular risk is ongoing.
The most common side effects of the drug include nasopharyngitis, upper respiratory tract infection, flu, back pain, and reactions such as redness, pain, or bruising where the injection is given. Allergic reactions, such as rash and hives, have been reported with the use of Repatha. Patients are advised to stop using Repatha and get medical help if they experience symptoms of a serious allergic reaction.