Long-term study shows promising results for evinacumab in treating HoFH

Evinacumab significantly reduces LDL cholesterol levels in patients with homozygous familial hypercholesterolaemia and is generally well tolerated, with sustained efficacy and safety over the long term for both adults and adolescents, according to a study.

This open-label, single-arm Phase 3 trial (NCT03409744) included 116 patients, with 102 adults and 14 adolescents. Participants, aged 12 years and older, received intravenous evinacumab at a dose of 15 mg/kg every 4 weeks, alongside their stable lipid-lowering therapy.

Patients were treated for a median duration of 104.3 weeks. There was a significant reduction in LDL cholesterol levels with evinacumab. On average, LDL-C decreased by 43.6% across the entire cohort by Week 24, with adults showing a 41.7% reduction and adolescents experiencing a 55.4% decrease.

The treatment was generally well tolerated. A majority of patients (80.2%) reported treatment-emergent adverse events, while serious treatment-emergent adverse events occurred in 23.3% of the cohort. Only 3 patients discontinued the treatment due to adverse events, none of which were linked to evinacumab. There were 2 reported deaths during the study period, but neither was attributed to the medication.

Reference
Gaudet D, Greber-Platzer S, Reeskamp LF, et al. Evinacumab in homozygous familial hypercholesterolaemia: long-term safety and efficacy. Eur Heart J. 2024;ehae325. doi: 10.1093/eurheartj/ehae325. Epub ahead of print. PMID: 38856678.