FDA Extends Approval Of Regeneron’s Evkeeza For Inherited High Cholesterol

The US FDA has extended the approval of Evkeeza (evinacumab-dgnb) to treat children aged 5 to 11 with homozygous familial hypercholesterolemia (HoFH), an inherited condition that causes dangerously high levels of cholesterol. 

Evkeeza was originally approved in February 2021 as an add-on therapy to assist other lipid-lowering drugs in patients aged 12 years and older with HoFH. 

The indication expansion makes Evkeeza the first angiopoietin-like 3 (ANGPTL3) inhibitor to help control severely high low-density lipoprotein cholesterol (LDL-C) in children as young as 5.

“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,” said Mary McGowan, M.D., Chief Medical Officer of the Family Heart Foundation, an organization dedicated to help families who live with heart disease.

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Evkeeza Lowers LDL-C by 48% from Baseline at Week 24

ANGPTL3 is a protein that slows certain enzymes that break down fats like LDL-C. By blocking ANGPTL3, Evkeeza allows faster breakdown of fats to control high cholesterol. 

The expanded approval is based on additional data from the pivotal trial of Evkeeza in HoFH. Despite treatment with other lipid-lowering therapies such as statins, children entered the trial with LDL-C levels more than twice the target. After receiving 15 mg/kg of Evkeeza every four weeks, patients achieved an average LDL-C reduction of 48% at week 24, meeting the primary endpoint. 

The drug also satisfied other secondary endpoints including levels of apolipoprotein B (ApoB), non-high-density lipoprotein cholesterol (non-HDL-C) and total cholesterol.

While the safety profile of Evkeeza in the trial was consistent with those in older groups, patients aged 5-11 may additionally experience fatigue. The most common adverse events included COVID-19, fever, headache, and throat pain, which were mostly mild or moderate, and did not lead to discontinuation. 

Regeneron is responsible for the development and marketing of Evkeeza in the US. The company partnered with Ultragenyx last January to distribute Evkeeza outside of the US. The deal included an $30 million upfront payment to Regeneron, while the company could receive up to $63 million in additional milestones. 

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Joy Lin

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