2023-03-23| Trials & Approvals

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

by Joy Lin
Share To

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.

Related Article: Regeneron Pays CytomX $2 Billion Biobucks to Develop Bispecific Cancer Therapies Together

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. 

© All rights reserved. Collaborate with us:
Related Post
ImmunityBio’s ANKTIVA® Granted FDA Approval: Breakthrough IL-15 Receptor Agonist First-in-Class for BCG-Unresponsive Non-Muscle Invasive Bladder Cancer
Wegovy Expands Its Reach: First Weight-Loss Medication Approved to Reduce Cardiovascular Risk
Groundbreaking CRISPR/Cas9-based Genome Editing Therapy Secured the Second FDA Approval
Preventive Medicine for Neurodegeneration on the Rise: New Breakthrough in Routine Blood Testing for Alzheimer’s Disease
Discrimination Leads to Rapid Aging, Science Shows the Detrimental Effects of this Act
A New Study has Linked Treatment Resistant Depression to Body Mass Index
AI Reveals Sex-Related Brain Differences and Precision Medicine Potential
The EU Sustainability Directive’s Influence on Pharmaceutical Supply Chains
New UC Berkeley Study Challenges Traditional Views on Lactate and Carbohydrate Metabolism
First Person to Receive Transplanted Pig Kidney has Died
Scroll to Top