Evinacumab reduces lipid levels in children with HoFH aged 5-11 years with LDL-C >130 mg/dL (3.4 mmol/L) despite optimised lipid lowering therapy (LLT), according to results of a Phase 3 open label study published in Circulation.
In the study of 14 patients, 5-11 years of age with genetically proven HoFH (true homozygotes and compound heterozygotes), evinacumab 15 mg/kg i.v. every four weeks decreased LDL-C by a mean -48.3% (±10.4%) from baseline to week 24. ApoB was reduced by -41.3% (±9.0%), non-HDL-C by -48.9% (±9.8%), and total cholesterol by -49.1% (±8.1%). Treatment-emergent adverse events (TEAEs) were reported in 10 (71.4%) patients, though only two (14.3%) reported events were considered to be treatment-related (nausea and abdominal pain). One serious TEAE (tonsillitis) was not considered treatment-related.
The authors concluded that evinacumab is a new treatment for these high risk, difficult-to-treat young patients with HoFH and inadequately controlled LDL-C despite optimised LLT. Approval of evinacumab by the US Food and Drug Administration (FDA) was extended to treat patients with HoFH aged 5-11 years in March 2023.
Wiegman A, Greber-Platzer S, Ali S et al. Evinacumab for pediatric patients with homozygous familial hypercholesterolemia. Circulation. 2023 Oct 20. Epub ahead of print.