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# Regeneron’s Evkeeza OK’d for Rare High Cholesterol
- URL: https://www.fdaweb.com/regenerons-evkeeza-okd-for-rare-high-cholesterol/
- Published: 2021-02-11T12:00:00.000Z
- Updated: 2026-09-14T16:46:23.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5148649

FDA has approved a Regeneron Pharmaceuticals BLA for Evkeeza (evinacumab-dgnb) as an adjunct to other low-density lipoprotein cholesterol (LDL-C) lowering therapies to treat adult and pediatric patients aged 12 years and older with homozygous familial hypercholesterolemia (HoFH). Evkeeza works by binding to and blocking the function of angiopoietin-like 3, a protein that plays a large role in lipid metabolism.

Approval is based on data from the Phase 3 ELIPSE HoFH trial that was [published](https://c212.net/c/link/?t=0&l=en&o=3066029-1&h=9852725&u=https%3A%2F%2Finvestor.regeneron.com%2Fnews-releases%2Fnews-release-details%2Fnew-england-journal-medicine-publishes-positive-phase-3&a=published&ref=fdaweb.com) 8/2020 in the *New England Journal of* Medicine. The trial met its primary endpoint, with Evkeeza-treated patients reducing their LDL-C from baseline by 49% on average compared to placebo at week 24 (47% reduction Evkeeza, 2% increase placebo).

The most common adverse reactions reported from the combined safety analysis of placebo-controlled trials after 24 weeks that occurred more frequently in Evkeeza patients than placebo were nasopharyngitis (16% Evkeeza, 13% placebo), influenza-like illness (7% Evkeeza, 6% placebo), dizziness (6% Evkeeza, 0% placebo), rhinorrhea (5% Evkeeza, 0% placebo), nausea (5% Evkeeza, 2% placebo), pain in extremity (4% Evkeeza, 0% placebo) and asthenia (4% Evkeeza, 0% placebo), the company says. Adverse reactions led to treatment discontinuation in 2% of patients treated with Evkeeza, including one case of anaphylaxis that resolved with treatment, and 2% of patients who received placebo, it says.