On August 27, the US Food and Drug Administration (FDA) approved Repatha (evolocumab) injection, marketed by Amgen Inc., of Thousand Oaks, California, for some patients who are unable to manage their low-density lipoprotein (LDL) cholesterol under control with current treatment options. Repatha is the second drug approved in a new class of drugs known as PCSK9 inhibitors; it is approved 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.
The FDA explains that familial hypercholesterolemia (encompassing both HeFH and HoFH) is an inherited disease that causes high levels of LDL cholesterol. A high level of LDL cholesterol in the blood is related to cardiovascular disease. Heart disease is the number one cause of death in the US for both men and women. According to the Centers for Disease Control and Prevention (CDC), approximately 610,000 individuals die of heart disease in the US––that factors out to one in every four deaths.
“Repatha provides another treatment option in this new class of drugs for patients with familial hypercholesterolemia or with known cardiovascular disease who have not been able to lower their LDL cholesterol enough with statins,” explained John Jenkins, M.D., director of the Office of New Drugs, Center for Drug Evaluation and Research. He added, “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.”
Repatha is an antibody that targets a specific protein, known as 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 cleanse LDL cholesterol from the blood and, as a result, lower LDL cholesterol levels.
The effectiveness and safety of Repatha were evaluated in one 52-week placebo-controlled trial and eight 12-week placebo-controlled trials in individuals with primary hyperlipidemia, including two that specifically enrolled participants with HeFH and one that enrolled participants with HoFH. In one of the 12-week trials, 329 participants with HeFH, who required additional lowering of LDL cholesterol despite statins with or without other lipid-lowering therapies, were randomily assigned to receive Repatha or a placebo for 12 weeks. The patients taking Repatha had an average reduction in LDL cholesterol of approximately 60%, compared to the placebo.
The most common side effects of Repatha include nasopharyngitis (inflammation of the nose and throat), 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. The FDA recommends that patients should stop using Repatha and seek medical help if they experience symptoms of a serious allergic reaction.