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A new class of lipid-lowering drugs for patients with a homozygous form of hypercholesterolemia


Evinacumab is a human monoclonal antibody inhibitor of angiopoietin-like 3, a liver protein that boosts levels of LDL cholesterol and triglycerides. Evinacumab, the first agent from a new class of lipid-lowering drugs, showed a "remarkable" and unprecedented level of LDL-cholesterol lowering in a pivotal trial with 65 patients with homozygous familial hypercholesterolemia. Monthly intravenous infusions of evinacumab cut LDL cholesterol levels by an average of 135 mg/dL from baseline, a 47% mean reduction, after 24 weeks of treatment in 43 homozygous familial hypercholesterolemia (HoFH) patients.  It was equally effective in the roughly one-third of patients with a minimal residual level of LDL receptor activity, which have "null/null" mutations.