The FDA has approved mipomersen (Kynamro – Genzyme) and lomitapide (Juxtapid – Aegerion), each in addition to a low-fat diet and other lipid-lowering medications, to reduce cholesterol levels in patients with homozygous familial hypercholesterolemia (HoFH).

Evolocumab (Repatha – Amgen), a subcutaneously injected PCSK9 inhibitor, has been approved by the FDA as an adjunct to diet and maximally tolerated statin therapy for adults with heterozygous familial hypercholesterolemia (HeFH) or clinical atherosclerotic cardiovascular disease who require additional lowering of LDL-cholesterol (LDL-C). It was also approved as an adjunct to diet and other LDL-lowering therapies in patients with homozygous familial hypercholesterolemia (HoFH) who require additional lowering of LDL-C. Evolocumab is the second PCSK9 inhibitor to be approved in the...

The National Cholesterol Education Program recommends that LDL-C be lowered to less than 100 mg/dL (2.6 mmol/L) and considers a value <70 mg/dL (1.8 mmol/L) a reasonable goal for patients at very high risk.

The results of the recently published FOURIER trial have shown a reduction in cardiovascular events with addition of the PCSK9 inhibitor evolocumab (Repatha) to statin therapy in patients with atherosclerotic cardiovascular disease (ASCVD).

The FDA has approved inclisiran (Leqvio – Novartis), a small interfering RNA (siRNA) directed to proprotein convertase subtilisin/kexin type 9 (PCSK9) mRNA, as an adjunct to diet and maximally tolerated statin therapy for subcutaneous (SC) treatment of adults with heterozygous familial hypercholesterolemia (HeFH) or clinical atherosclerotic cardiovascular disease (ASCVD) who require additional lowering of low-density lipoprotein cholesterol (LDL-C). Inclisiran is the first FDA-approved PCSK9-directed siRNA therapeutic agent.

The FDA has announced changes in the labeling of simvastatin to reduce the risk of myopathy. These changes include limiting the use of the 80-mg maximum dose to patients who have been taking it for 12 months or more without evidence of myopathy and new recommendations for use of simvastatin with other drugs. Simvastatin is available alone (Zocor, and others) and in combination with ezetimibe (Vytorin) and with niacin (Simcor).

The FDA has approved lerodalcibep (Lerochol – Lib Therapeutics), a subcutaneously injected proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor, to reduce low-density lipoprotein cholesterol (LDL-C) in adults with hypercholesterolemia, including heterozygous familial hypercholesterolemia (HeFH). Lerodalcibep is the fourth subcutaneously injected PCSK9-directed therapy to be approved in the US; the monoclonal antibodies alirocumab (Praluent) and evolocumab (Repatha) and the small interfering RNA inclisiran (Leqvio) were approved earlier (see Table...

The results of the AIM-HIGH trial conducted by the US National Heart, Lung and Blood Institute (NHLBI) were recently published. The goal of the trial was to test whether addition of niacin to intensive statin therapy would further reduce the risk of cardiovascular disease. The trial was stopped prematurely after an average follow-up of 3 years because niacin therapy had not shown any clinical benefit.

The recent "PROVE IT" trial in patients with coronary heart disease showed clinical benefits associated with reducing LDL cholesterol concentrations lower than the 100 mg/dL (2.59 mmol/L) or less that had been considered optimal.