19 Sept 2023 • Patients with severe hypercholesterolemia who are unresponsive to a number of lipid-lowering therapies, including a PCSK9 inhibitor and high-dose statin, can have a significant reduction in LDL-cholesterol levels when treated longer-term with evinacumab, a new open-label study shows. The new study, which is published this week in JAMA Cardiology, focuses on patients with primary hypercholesterolemia refractory to lipid-lowering therapy.

LDL levels were cut nearly in half from baseline and more than 50% of patients got to the recommended treatment target of less than 70 mg/dL, report investigators, while one-third got down below 55 mg/dL. In terms of safety, the most common side effect was nasopharyngitis (12.5%), but no treatment-emergent adverse events led to deaths or stopping treatment.

Evinacumab is a fully human monoclonal antibody inhibitor of angiopoietin-like protein 3 (ANGPTL3), which plays a role in regulating lipid metabolism. It was approved in 2021 by the US Food and Drug Administration as a breakthrough therapy for patients with homozygous FH (HoFH), including pediatric patients. It’s administered by weight (15 mg/kg).

“Of patients with heterozygous familial hypercholesterolemia, which was about three-quarters of our population—they had clinical characteristics and genetically confirmed familial hypercholesterolemia—about 20% still have an LDL-cholesterol level above the minimally acceptable targets,” lead investigator Robert Rosenson, MD, says “The reason that it's so important to achieve the targets is that these individuals had an elevated LDL cholesterol before they were born and we know that the onset of coronary heart disease begins early.”

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