LAUSR

Many patients with homozygous familial hypercholesterolaemia (FH) do not reach guideline­ recommended levels of LDL cho­ lesterol (LDL­ C) with currently available therapies. A phase III trial now shows that treatment with evinacumab, a monoclonal antibody against angiopoietin­ related protein 3 (ANGPTL3), reduces LDL­ C levels by nearly 50% compared with placebo in patients with homozygous FH receiving maximum doses of lipid­ lowering therapy with or without apheresis, regardless of their LDL­ receptor functional degree. The evinacumab group had a 47.1% reduction in LDL­ C levels at 24 weeks from baseline com­ pared with a 1.9% increase in the placebo group (between­ group difference −49.0 percentage points, P < 0.001). LDL­ C levels were lower with evinacumab than with placebo in patients with null–null LDLR variants (−43.4% versus + 16.2%) or with non­ null LDLR variants (−49.1% versus −3.8%). Evinacumab also induced a 30% decrease in HDL­ cholesterol levels compared with placebo.