Evinacumab in homozygous familial hypercholesterolaemia: long-term safety and efficacy.

ABSTRACT:

Background and aims

Homozygous familial hypercholesterolaemia (HoFH) is a rare genetic disorder characterized by severely elevated LDL cholesterol (LDL-C) and premature atherosclerotic cardiovascular disease. In the pivotal Phase 3 HoFH trial (NCT03399786), evinacumab significantly decreased LDL-C in patients with HoFH. This study assesses the long-term safety and efficacy of evinacumab in adult and adolescent patients with HoFH.

Methods

In this open-label, single-arm, Phase 3 trial (NCT03409744), patients aged ≥12 years with HoFH who were evinacumab-naïve or had previously received evinacumab in other trials (evinacumab-continue) received intravenous evinacumab 15 mg/kg every 4 weeks with stable lipid-lowering therapy.

Results

A total of 116 patients (adults: n = 102

SUBMITTER: Gaudet D 

PROVIDER: S-EPMC11242450 | biostudies-literature | 2024 Jul

REPOSITORIES: biostudies-literature