The U.S. Food and Drug Administration (FDA) today approved evolocumab injection for some patients who are unable to get their LDL-c under control with current treatment options.
Evolocumab, the second drug approved in a new class of drugs known as PCSK9 inhibitors, 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 efficacy and safety of evolocumab were evaluated in one 52-week placebo-controlled trial and eight 12-week placebo-controlled trials in participants with primary hyperlipidaemia, including two that specifically enrolled participants with HeFH and one that enrolled participants with HoFH. In one 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 evolocumab had an average reduction in LDL-c of approximately 60 percent, compared to placebo.
The most common side effects of evolocumab 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 evolocumab. Patients should stop using evolocumab and get medical help if they experience symptoms of a serious allergic reaction. A trial evaluating the effect of adding evolocumab to statins for reducing cardiovascular risk is ongoing.