FDA approval for evolocumab


The FDA has approved evolocumab (Repatha, Amgen), which is a new cholesterol-lowering medication 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). The drug is indicated as an adjunct to diet and maximally tolerated statin therapy for the treatment of adults with heterozygous familial hypercholesterolemia or clinical atherosclerotic cardiovascular disease who require additional lowering of LDL-C.

According to a press release, the drug is also indicator as an adjunct to diet and other LDL-lowering therapies for the treatment of patients with homozygous familial  hypercholesterolemia who require additional lowering of LDL-C. The press release also notes that the effect of evolocumab on cardiovascular morbidity and mortality has not been determined.

In phase 3 trials, adding evolocumab to background lipid-lowering therapy that included statins resulted in intensive reductions in LDL-C levels with favourable effects on other lipid parameters. In patients with clinical atherosclerotic cardiovascular disease or heterozygous familial hypercholesterolemia, evolocumab reduced LDL-C by approximately 54– 77% compared with placebo. In a pivotal phase 3 trial, 90% of clinical atherosclerotic cardiovascular disease patients who received evolocumab in addition to maximum doses of statins achieved a LDL-C level less than 70mg/dL. In patients with homozygous familial hypercholesterolemia, evolocumab reduced LDL-C by approximately 30% compared with placebo.

Evolocumab is generally well-tolerated with an established safety profile. The most common adverse reactions that occurred in greater than 5% of the evolocumab group, and more frequently than in the placebo group, were nasopharyngitis, upper respiratory tract infection, influenza, back pain and injection site reactions.

Evolocumab is available as a single-use 140 mg prefilled SureClick autoinjector or prefilled syringe that patients can self-administer at the recommended dose for adults of 140mg every two weeks or 420 mg once a month. For adults with homozygous familial hypercholesterolemia, the recommended dose is 420mg once a month. Amgen will continue discussions with the FDA regarding the 420mg every two weeks dosing for heterozygous familial hypercholesterolemia patients.

Sean E Harper, executive vice president of Research and Development at Amgen, says: “Data from key clinical studies have shown that evolocumab significantly reduces LDL cholesterol in patients who have not been able to lower their LDL cholesterol through diet and statins alone. At Amgen, we are committed to improving the lives of patients and are inspired by the potential for evolocumab to aid in the global fight against one of the major risk factors for cardiovascular disease.”