Evinacumab for Homozygous Familial Hypercholesterolemia.
Frederick J RaalRobert S RosensonLaurens F ReeskampJohn J P KasteleinPaolo RubbaShazia AliPoulabi BanerjeeKuo-Chen ChanDaniel A GipeNagwa KhillaRobert PordyDavid M WeinreichGeorge D YancopoulosYi ZhangDaniel Gaudetnull nullPublished in: The New England journal of medicine (2020)
In patients with homozygous familial hypercholesterolemia receiving maximum doses of lipid-lowering therapy, the reduction from baseline in the LDL cholesterol level in the evinacumab group, as compared with the small increase in the placebo group, resulted in a between-group difference of 49.0 percentage points at 24 weeks. (Funded by Regeneron Pharmaceuticals; ELIPSE HoFH ClinicalTrials.gov number, NCT03399786.).