FDA extends treatment for hypercholesterolemia to young children

FDA extended1 Regeneron’s Evkeeza (evinacumab-dgnb) has been approved as an adjunct to other lipid-lowering therapies for the treatment of children aged 5 to 11 years with homozygous familial hypercholesterolemia (HoFH). HoFH is a very rare genetic disorder characterized by very high levels of LDL-C, and until now there have been limited treatment options to help reduce LDL-C in young children. It affects approximately 1,300 people in the United States and is the most severe form of familial hypercholesterolemia. HoFH patients are at risk for early atherosclerosis and heart disease, even in their teens.

Evkeeza is a monoclonal antibody that blocks the function of angiopoietin-like 3 (ANGPTL3) and is indicated in children as young as 5 years to control high levels of low-density lipoprotein cholesterol. Evkeeza was first approved in 2021 as an adjunct to other lipid-lowering therapies in people 12 years and older.

“Adding Evkeeza to standard lipid-lowering therapy in this pivotal trial was able to reduce LDL-C in children, with the majority achieving a reduction of nearly 50%.” Carissa M. Baker-Smith, MD, MPH, co-director of the Nemours Cardiac Center Cardiovascular Research and Innovation Program, director of preventive cardiology, pediatric cardiologist and principal investigator of the Nemours Cardiac Center Pediatric, said in a press release.

Wholesale purchase costs for Evkeeza are $11,661 for 345 mg/2.3 mL single dose vials and $40,560 for 1200 mg/8 mL single dose vials.spokesperson said prescription watch Since this is a weight-based drug and is competitively priced in line with other ultraorphan biologics, the actual cost will vary.

Regeneron offers a co-payment program for patients with commercial insurance that provides up to $25,000 in assistance annually. We also have a patient assistance program for patients without insurance.

Label extension is based on the results of a phase 3 trial in 20 children aged 5 to 11 years. The addition of Evkeeza allowed the child to reduce LDL-C by an average of 48% at her 24th week, meeting the trial’s primary endpoint. Significant reductions were also observed in other key secondary endpoints, including levels of apolipoprotein B (ApoB), non-high-density lipoprotein cholesterol, and total cholesterol.

The observed safety profile of Evkeeza was consistent with previous results, with the added side effect of fatigue. Fatigue was reported in 3 of her patients. The most common adverse events were COVID-19, fever, headache, sore throat, upper abdominal pain, diarrhea, vomiting, fatigue, nasopharyngitis, rhinitis, and cough. Most reported adverse events were mild or moderate in severity and none led to study discontinuation.

This article originally appeared in Formulary Watch.


1. FDA approves first-in-class evkeeza® (evinacumab-dgnb) for infants with ultra-rare form of high cholesterol. press release. March 22, 2023. Accessed March 23, 2023.

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