FDA approves Repatha (evolocumab) from Amgen to treat LDL cholesterol

The FDA approved Repatha (evolocumab) injection from Amgen for some patients who are unable to get their low-density lipoprotein (LDL) cholesterol under control with current treatment options. Repatha, 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 Repatha were evaluated in one 52-week placebo-controlled trial and eight 12-week placebo-controlled trials in participants with primary hyperlipidemia, 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 Repatha had an average reduction in LDL cholesterol of approximately 60 percent, compared to placebo.