March 22, 2023

1 min read

Disclosures: Baker-Smith reports serving as a site primary investigator for the phase 3 trial of evinacumab-dgnb in pediatric patients with HoFH and receiving research grants from Regeneron.

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Regeneron Pharmaceuticals announced the FDA has extended approval of evinacumab-dgnb as an adjunct to other lipid-lowering therapy for the treatment of homozygous familial hypercholesterolemia in children aged 5 to 11 years.

After priority review by the FDA, evinacumab-dgnb (Evkeeza) is the first angiopoietin-like 3 inhibitor treatment to be indicated for children as young as 5 years old to treat high LDL from homozygous familial hypercholesterolemia (HoFH), according to a press release from the company.

A Healio previously reported, evinacumab-dgnb was approved in February 2021 as an adjunct to other lipid-lowering therapies for patients aged 12 years or older with HoFH.

The extended approval is based on results of a phase 3 trial that enrolled 20 pediatric patients with a mean LDL level of 264 mg/dL. The addition of evinacumab-dgnb was associated with an average LDL reduction of 48% at week 24, meeting the trials primary endpoint, according to the release.

Reductions were also observed in apolipoprotein B, non-HDL and total cholesterol, and the safety profile of evinacumab-dgnb in children aged 5 to 11 years was consistent with the safety profile observed in adults and pediatric patients aged 12 years or older, with the additional adverse reaction of fatigue reported in 15% of patients, according to the release.

Carissa M. Baker-Smith

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, Carissa M. Baker-Smith, MD, MPH, director of pediatric preventive cardiology, Nemours Childrens Health, Delaware Valley, said in the release. 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.

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FDA extends approval of evinacumab for HoFH to children aged 5 to ... - Healio