FDA extends approval of Evkeeza for homozygous familial hypercholesterolemia in young children


Evkeeza is the first angiopoietin-like 3 (ANGPTL3) inhibitor indicated for patients as young at 5 years to control high levels of low-density lipoprotein cholesterol (LDL-C) as a result of HoFH.

The FDA has extended the approval of Evkeeza (evinacumab-dgnb; Regeneron) as an adjunct treatment to other lipid-lowering therapies for homozygous familial hypercholesterolemia (HoFH) in children aged 5 to 11 years, according to a recent Regeneron press release announcing the approval.

Previously, Evkeeza was FDA approved as an adjunct in HoFH patients aged 12 years and older. Now, with this most recent approval, Evkeeza is the first angiopoietin-like 3 (ANGPTL3) inhibitor indicated for patients as young at 5 years to control high levels of low-density lipoprotein cholesterol (LDL-C) as a result of HoFH.

“At the Family Heart Foundation, we know that children with homozygous familial hypercholesterolemia, and those caring for them, often live in fear of what the future holds as they contend with the dangerously high levels of bad cholesterol, or LDL-C, caused by this genetic disorder,” said Mary McGowan, MD, chief medical officer of the Family Heart Foundation. “Only 5% of rare diseases actually have an FDA-approved treatment. With this FDA approval, the HoFH community now has a much-needed treatment for young children, potentially making it possible for many to achieve recommended LDL-C levels much earlier in the course of this rare disease. This is a hopeful development for those living with HoFH.”

This approval is backed by positive data from a 3-part, single-arm, open-label trial evaluating Evkeeza as an adjunct to other lipid-lowering therapies. Patients entered the study with a more than double average LDL-C level, 264 mg/dL vs a target of less than 110 mg/dL. When administered Evkeeza, the patients’ LDL-C levels were reduced by 48% at week 24 on average, thus, achieving the study’s primary endpoint. Additionally, investigators observed significant reductions in key secondary endpoints such as levels of apolipoprotein B (ApoB), non-high-density lipoprotein cholesterol (non-HDL-C) and total cholesterol.

Evkeeza’s safety profile was also consistent with that of adults and pediatric patients 12 years and older; however, investigators also recorded an additional adverse reaction of fatigue, occurring in 15% (n = 3) of patients.

In the study, the most common adverse events (AEs) occurred in more than 15% of patients included COVID-19 (n = 15), pyrexia (n = 5), headache (n = 4), throat pain (oropharyngeal pain, n = 4) and upper abdominal pain (n = 3), diarrhea (n = 3), vomiting (n = 3), fatigue (n = 3), nasopharyngitis (n = 3), rhinitis (n = 3), and cough (n = 3).

“Guidelines recommend screening all children at high risk for homozygous familial hypercholesterolemia starting at age 2. However, until now, a positive diagnosis was often met with the frustration of having limited treatment options to help these children,” said Carissa M. Baker-Smith, MD, MPH, codirector of Nemours Cardiac Center Cardiovascular Research and Innovation Program, Director of Nemours Cardiac Center Pediatric Preventive Cardiology, pediatric cardiologist, and a trial investigator. “By adding Evkeeza to standard lipid-lowering therapies in this pivotal trial, children were able to reduce their LDL-C, with the vast majority able to achieve declines of nearly 50%. These are clinically meaningful results that physicians should consider when developing a treatment approach for these young patients.”


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

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