FDA Approves Evinacumab (Evkeeza) for HoFH in Children As Young As 1 Year Old

The US Food and Drug Administration has approved Regeneron Pharmaceuticals’ evinacumab-dgnb (Evkeeza) angiopoietin-like 3 (ANGPTL3) antibody as an adjunct to diet and exercise and other lipid-lowering therapies for the treatment of children 1 to < 5 years of age with homozygous familial hypercholesterolemia (HoFH).1

“The approval of [evinacumab-dgnb] for children as young as 1 year of age addresses a critical unmet need for those with homozygous familial hypercholesterolemia, a life-threatening condition that causes extraordinarily high LDL-C levels from birth,” Katherine Wilemon, founder and CEO of the Family Heart Foundation, said in a statement.1 “Families and their medical teams will now have an effective treatment option for these very young children who are at risk of serious complications from diseased arteries and calcified valves without timely and sufficient LDL-C lowering. This development underscores the importance of, and urgency needed in, identifying children with FH through pediatric screenings in accordance with guidelines.”

Evinacumab-dgnb was developed using Regeneron’s VelocImmune® technology and is a fully human monoclonal antibody that binds to and blocks the function of ANGPTL3, a protein that inhibits lipoprotein lipase (LPL) and endothelial lipase (EL) and regulates circulating lipids, including LDL-C.1

It was initially approved in 2021 for adults and adolescents aged 12 years and older with HoFH based on results of the phase 3 ELIPSE HoFH trial. A 24-week, 65-patient trial, results of the study indicated use of evinacumab was associated with a 49% reduction in LDL-C from baseline relative to placebo therapy (P <.0001). Further analysis of the trial indicated use was also associated with significant reductions in levels of apolipoprotein B, non-high-density lipoprotein cholesterol, and total cholesterol, compared to placebo (P <.0001 for all).1,2

It was later approved for children 5-11 years of age in 2023, making it the first ANGPTL3 inhibitor treatment indicated for children as young as 5 years old to lower elevated LDL-C caused by HoFH based on data from a 3-part, single-arm trial conducted in this age group.1,2

Among the 20 patients included Part A and B of the trial, the observed safety profile was consistent with previous data in adults and pediatric patients aged 12 years and older. Adverse events occurring among more than 15% of patients were COVID-19 (n = 15), pyrexia (n = 5), headache (n = 4), oropharyngeal pain (n = 4) as well as upper abdominal pain, diarrhea, vomiting, fatigue, nasopharyngitis, rhinitis and cough (n = 3). None of the adverse events led to study discontinuation.2

The extended indication for evinacumab-dgnb was supported by clinical efficacy and safety data among 6 children living with HoFH, including pharmacokinetic data among 4 of these patients, who took part in either the US expanded access program or ex-US compassionate use program for evinacumab-dgnb. No new safety concerns have been identified in the compassionate use program. The most common adverse reactions (≥5%) include nasopharyngitis, influenza-like illness, dizziness, rhinorrhea, nausea, and fatigue.1

“[Evinacumab-dgnb] is a testament to the power of Regeneron’s science and proprietary technologies in developing first-in-class, lifechanging medicines that become standard-of-care,” said George Yancopoulos, MD, PhD, board co-chair, president and chief scientific officer at Regeneron.1 “This label extension adds to our growing commitment to the rare disease space, which includes diverse clinical development programs in neuromuscular and genetic diseases – such as myasthenia gravis, otoferlin hearing loss, and fibrodysplasia ossificans progressiva – for which we’ve shared data.”

References

1. Regeneron. Evkeeza® (evinacumab-dgnb) ANGPTL3 Antibody Approved in the U.S. for Children as Young as 1 Year Old with Ultra-Rare Form of High Cholesterol. September 26, 2025. Accessed September 26, 2025. https://investor.regeneron.com/news-releases/news-release-details/evkeezar-evinacumab-dgnb-angptl3-antibody-approved-us-children

2. Campbell P. FDA Approves Evinacumab for HoFH in Children Aged 5-11 Years Old. HCPLive. March 22, 2023. Accessed September 26, 2025. https://www.hcplive.com/view/fda-approves-evinacumab-for-hofh-in-children-aged-5-11-years-old