FDA Extends Evkeeza Indication to Include Young Children

The FDA has extended the approval of Regeneron’s Evkeeza (evinacumab-dgnb) as an adjunct to other lipid-lowering therapies to treat children aged 5 to 11 with homozygous familial hypercholesterolemia (HoFH). HoFH is an ultra-rare genetic condition characterized by extremely high levels of LDL-C and, until now, treatment options to help reduce LDL-C in younger children have been limited. It affects about 1,300 people in the United States and is the most severe form of familial hypercholesterolemia. Those living with HoFH are at risk for premature atherosclerotic disease and cardiac events even in their teenage years.

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

Carissa M. Baker-Smith, M.D.

“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,” Carissa M. Baker-Smith, M.D., MPH, co-director of Nemours Cardiac Center Cardiovascular Research and Innovation Program, director of Nemours Cardiac Center Pediatric Preventive Cardiology, pediatric cardiologist, and a trial investigator, said in a press release.

Evkeeza has a wholesale acquisition cost of $11,661 for single-dose vial for 345 mg/2.3 mL and $40,560 for a single-dose vial for 1200mg/8 mL. A spokesperson told Formulary Watch that the actual cost will vary as this is a weight-based medication and has a competitive price that is in line with other ultra-orphan biologics.

Regeneron offers a copay program for commercially insured patients that provides up to $25,000 in assistance per year and also has a patient assistance program for patients without insurance.

The label extension was based on phase 3 trial results of 20 children aged 5 to 11. With the addition of Evkeeza, children were able to reduce their LDL-C by 48% at week 24 on average, 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 safety profile of Evkeeza observed was consistent with previous results, with the additional adverse reaction of fatigue. Fatigue was reported in three patients. The most common adverse events were COVID-19, fever, headache, throat pain, as well as upper abdominal pain, diarrhea, vomiting, fatigue, nasopharyngitis, rhinitis and cough. Most reported AEs were mild or moderate, and none led to study discontinuation.