The US Food and Drug Administration (FDA) has expanded the approval of Regeneron Pharmaceuticals’ fully human monoclonal antibody, Evkeeza (evinacumab-dgnb), for the treatment of homozygous familial hypercholesterolemia (HoFH) in children aged between one and four.
The decision enables the Evkeezaangiopoietin-like 3 (ANGPTL3) antibody to be used as an adjunct with dietary adjustments, exercise and other lipid-lowering therapies.
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HoFH is the most extreme form of familial hypercholesterolemia.
The decision to broaden the antibody’s indication was based on clinical efficacy and safety data from six children with HoFH who participated in either the US expanded access programme or the compassionate use programme outside the US.
The compassionate use programme did not reveal any new safety issues.
Regeneron chief scientific officer, board co-chair and president George Yancopoulos stated: “Evkeeza is a testament to the power of Regeneron’s science and proprietary technologies in developing first-in-class, life-changing medicines that become standard-of-care.”
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By GlobalData“This label extension adds to our growing commitment to the rare disease space, which includes diverse clinical development programmes in neuromuscular and genetic diseases such as myasthenia gravis, otoferlin hearing loss and fibrodysplasia ossificans progressiva, for which we’ve shared data.”
The antibody initially received the US regulator’s approval in 2021 for HoFH patients aged 12 years and above after a placebo-controlled study demonstrated its ability to halve LDL-C (low-density lipoprotein cholesterol) levels when used alongside standard lipid-lowering treatments.
In 2023, the antibody’s approval was extended to cover children aged five to 11.
Evkeeza operates by binding to and inhibiting the ANGPTL3 protein that regulates lipids such as LDL-C in the bloodstream.
The antibody was invented leveraging the VelocImmune technology of Regeneron.
Regeneron oversees the development and distribution of the antibody within the US. The company is also partnering Ultragenyx to manage the clinical development, commercialisation and distribution of the antibody outside the US.
In June 2025, the FDA approved Sanofi and Regeneron’s Dupixent as a treatment option for adults with bullous pemphigoid, a rare skin-blistering condition.
