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# Regeneron Files sBLA for Evkeeza in Young Children
- URL: https://www.fdaweb.com/regeneron-files-sbla-for-evkeeza-in-young-children/
- Published: 2022-11-30T12:00:00.000Z
- Updated: 2026-09-14T18:08:05.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5153285

FDA has accepted for priority review a Regeneron Pharmaceuticals supplemental BLA for Evkeeza (evinacumab-dgnb) as an adjunct to other lipid-lowering therapies to treat children aged 5 to 11 years with homozygous familial hypercholesterolemia (HoFH). The agency has set a user fee review action date of 3/30/2023.

HoFH is a rare inherited condition that affects about 1,300 patients in the U.S. It occurs when two copies of the FH-causing genes are inherited, one from each parent, which leads to dangerously high levels of low-density lipoprotein-cholesterol (LDL-C or bad cholesterol), according to the company.

The submission is based on data from a trial evaluating Evkeeza’s efficacy in 14 children. Regeneron says the trial met its primary endpoint, showing children who added Evkeeza to other lipid-lowering therapies reduced their bad cholesterol by 48% at week 24.

Evkeeza is described as the first angiopoietin-like 3 targeted therapy [approved](https://c212.net/c/link/?t=0&l=en&o=3723238-1&h=1842251563&u=https%3A%2F%2Finvestor.regeneron.com%2Fnews-releases%2Fnews-release-details%2Ffda-approves-first-class-evkeezatm-evinacumab-dgnb-patients%2F&a=approved&ref=fdaweb.com) by FDA as an adjunct therapy for patients aged 12 years and older with HoFH.