Key clinical point: Evinacumab showed unprecedented LDL-lowering efficacy and safety for homozygous familial hypercholesterolemia patients in a pivotal trial.
Major finding: Twenty-four weeks of evinacumab treatment cut LDL cholesterol by 49%, compared with placebo.
Study details: A multicenter, placebo-controlled pivotal trial with 65 patients.
Disclosures: The study was sponsored by Regeneron, the company developing evinacumab and which is partially owned by Sanofi. Dr. Raal has received personal fees and/or research funding from Regeneron, Sanofi Aventis, Amgen, and The Medicines Company. Dr. Goldberg has received research funding and/or consulting fees from Regeneron and Sanofi, Akcea, Amarin, Amgen, Esperion, Ionis, Merck, Novartis, and Pfizer. Dr. Blom has been a consultant to and/or received research funding from Regeneron, Sanofi, Aegerium, Akcea, Amgen, Amryt, AstraZeneca, Eli Lilly, Esperion, Gemphire, MSD, and Novo Nordisk. Dr. Bhatt has received research funding from many companies including Regeneron and Sanofi.
Raal F. ACC 20. Abstract 411-12.