Regeneron reports evinacumab phase 3 results for children 5 to 11 years old with HoFH


Evinacumab phase 3 results show a 48% LDL-C reduction in children with homozygous familial hypercholesterolemia.

Regeneron Pharmaceuticals recently announced positive results from a pivotal phase 3 trial evaluating evinacumab (Evkeeza; Regeneron) in children aged 5 to 11 years with homozygous familial hypercholesterolemia (HoFH).1 The results were presented at the 5th European Atherosclerosis Society Pediatric Familial Hypercholesterolemia symposium.

HoFH is categorized as a rare inherited disorder resulting in “extremely elevated low-density lipoprotein cholesterol levels and premature atherosclerotic cardiovascular disease (ASCVD),” according to The Lancet.2

The study met its primary end point, showing children who added evinacumab to other lipid-lowering therapies reduced low-density lipoprotein-cholesterol (LDL-C) by 48% on average at week 24, according to a press release announcing the results.1

Study participants entered the trial with reported dangerously high LDL-C (264 mg/dL on average) and 79% saw their LDL-C reduced by at least half with an absolute 132 mg/dL reduction in LDL-C on average from baseline.

“Despite treatment with other lipid-lowering therapies, children (n = 14) entered the trial with an average LDL-C level of 264 mg/dL, more than twice the target (<130 mg/dL) for pediatric patients with HoFH. After 24 weeks of [evinacumab] treatment (15 mg/kg every 4 weeks delivered intravenously [IV]), the phase 3 trial met its primary endpoint,” according to the Regeneron release.

Investigators saw reductions in levels of all lipid end point parameters assessed—generally observed with the first 8-weeks of treatment—including apolipoprotein B, non-high-density lipoprotein cholesterol, lipoprotein(a), and total cholesterol.

The most common adverse events (AEs) were oropharyngeal pain (21%), upper abdominal pain, diarrhea, headache, and nasopharyngitis (all 14%). Two severe AEs reported were aortic stenosis and tonsilitis, but both were considered unrelated to treatment.

Trial results will form the basis of a US Food and Drug Administration (FDA) regulatory submission later this year.


  1. Evkeeza (evinacumab) phase 3 trial demonstrates 48% LDL-C reduction in children with ultra-rare form of high cholesterol. Regeneron Pharmaceuticals. Published May 21, 2022. Accessed May 23, 2022.
  2. Tromp TR, Hartgers ML, Hovingh GK, et al. Worldwide experience of homozygous familial hypercholesterolaemia: retrospective cohort study. The Lancet. 2022;399(10326):719-728.
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