FDA Expands Evinacumab-dgnb Approval for Children as Young as 1 Year With HoFH

Regeneron Pharmaceuticals, Inc., announced FDA approval of evinacumab-dgnb (Evkeeza) for children as young as 1 year of age with homozygous familial hypercholesterolemia (HoFH), expanding treatment options for this rare and life-threatening condition marked by extremely high low-density lipoprotein cholesterol (LDL-C) levels from birth.¹

“The approval of Evkeeza 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,” said Katherine Wilemon, founder and CEO of the Family Heart Foundation, in a statement. “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 [familial hypercholesterolemia] through pediatric screenings in accordance with guidelines.”

In February 2021, the FDA granted its first approval for evinacumab-dgnb as an adjunct to other lipid-lowering therapies for adults and children aged 12 years and older with HoFH.² This approval was based on the pivotal phase 3 ELIPSE HoFH (NCT03399786) trial, which demonstrated that evinacumab-dgnb reduced LDL-C levels by nearly 50% at 24 weeks compared with placebo. As the first FDA-approved angiopoietin-like 3 (ANGPTL3) inhibitor, evinacumab-dgnb introduced a novel approach to treating patients with this condition.

Building on this progress, in March 2023 the FDA expanded evinacumab-dgnb’s indication to children aged 5 to 11 years with HoFH.³ This approval followed a pivotal pediatric trial showing that adding evinacumab-dgnb to standard lipid-lowering therapies resulted in a 48% mean reduction in LDL-C at 24 weeks. The expansion marked the first time an ANGPTL3 inhibitor was indicated for children as young as 5 years of age, offering earlier intervention for young patients facing lifelong risks of premature atherosclerotic disease and cardiac events.

The most recent FDA expanded approval further broadens evinacumab-dgnb to include children from age 1 year to less than 5 years with HoFH, providing families and clinicians with an effective treatment option for the youngest patients without introducing new safety concerns.¹ The most common adverse reactions included nasopharyngitis, influenza-like illness, dizziness, rhinorrhea, nausea, and fatigue.

With this latest approval, evinacumab-dgnb now offers a continuum of care for children of nearly all ages living with HoFH, enabling earlier and more sustained LDL-C control to help reduce long-term cardiovascular risk.

“Evkeeza is a testament to the power of Regeneron’s science and proprietary technologies in developing first-in-class, life-changing medicines that become standard-of-care,” said George D. Yancopoulos, MS, PhD, board co-chair, president, and chief scientific officer at Regeneron, in a statement. “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. Evkeeza (evinacumab-dgnb) ANGPTL3 antibody approved in the US for children as young as 1 year old with ultra-rare form of high cholesterol. News release. Regeneron. September 26, 2025. Accessed September 26, 2025. https://newsroom.regeneron.com/news-releases/news-release-details/evkeezar-evinacumab-dgnb-angptl3-antibody-approved-us-children

2. FDA approves first-in-class Evkeeza (evinacumab-dgnb) for patients with ultra-rare inherited form of high cholesterol. News release. Regeneron. February 11, 2021. Accessed September 26, 2025. https://investor.regeneron.com/news-releases/news-release-details/fda-approves-first-class-evkeezatm-evinacumab-dgnb-patients/

3. FDA approves first-in-class Evkeeza (evinacumab-dgnb) for young children with ultra-rare form of high cholesterol. News release. Regeneron. March 22, 2023. Accessed September 26, 2025. https://investor.regeneron.com/news-releases/news-release-details/fda-approves-first-class-evkeezar-evinacumab-dgnb-young-children