Evinacumab for Pediatric Patients With Homozygous Familial Hypercholesterolemia.

ABSTRACT:

Background

Homozygous familial hypercholesterolemia (HoFH) is a rare genetic disorder characterized by severely elevated low-density lipoprotein cholesterol (LDL-C) levels due to profoundly defective LDL receptor (LDLR) function. Given that severely elevated LDL-C starts in utero, atherosclerosis often presents during childhood or adolescence, creating a largely unmet need for aggressive LDLR-independent lipid-lowering therapies in young patients with HoFH. Here we present the first evaluation of the efficacy and safety of evinacumab, a novel LDLR-independent lipid-lowering therapy, in pediatric patients with HoFH from parts A and B of a 3-part study.

Methods

The phase 3, part B, open-label study treated 14 patients 5 to 11 years of age with genetically proven HoFH (true hom

SUBMITTER: Wiegman A 

PROVIDER: S-EPMC10814999 | biostudies-literature | 2024 Jan

REPOSITORIES: biostudies-literature