Conference Roundup
Homozygous Familial Hypercholesterolemia (HoFH)

Study demonstrates long-term efficacy of evinacumab in children with HoFH

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A long-term study presented at the 2024 National Lipid Association Scientific Sessions showed “substantial and durable” reductions in low-density lipoprotein cholesterol (LDL-C) in pediatric patients with homozygous familial hypercholesterolemia (HoFH) who were treated with evinacumab.

The study enrolled patients aged 5 to 11 years with an LDL-C >130 m/dL who were on lipid-lower therapies into 3 clinical trial cohorts:

  • Part A: phase 1b, 16-week study that included 6 patients who received evinacumab 15 mg/kg intravenously (IV)
  • Part B: phase 3, open-label, 24-week trial that included 14 patients who had not participated in part A
  • Part C: phase 3, open-label, 48-week extension study with an optional 24-week follow-up period that included all 20 patients from parts A and B who received evinacumab 15 mg/kg IV every 4 weeks

The pooled data presented at the meeting included participants from parts B and C for a total treatment duration of 72 weeks.

Mean LDL-C at baseline was 301.9 mg/dL. Evinacumab therapy reduced mean LDL-C by 44.6% from baseline to 48 weeks (n=16) and by 40.7% from baseline to 72 weeks (n=14).

Evinacumab also induced reductions in mean apolipoprotein B, non-high-density lipoprotein cholesterol, total cholesterol, and median lipoprotein a at both weeks 48 and 72.

Treatment-related adverse events occurred in all patients, with 2 patients experiencing serious events (tonsilitis and aortic valve stenosis), which the authors deemed “unrelated” to the study drug. No patients discontinued treatment or died during the reporting period.

The study was funded by Regeneron Pharmaceuticals, Inc.


Greber-Platzer S, Ali S, Waldron A, et al. The long-term efficacy and safety of evinacumab in pediatric patients with homozygous familial hypercholesterolemia. Abstract 186. Presented at the 2024 National Lipid Association Scientific Sessions, May 30-June, Las Vegas, Nevada.