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Regeneron's Evkeeza gains FDA approval for treating young children with homozygous familial hypercholesterolemia, addressing a critical health need.
Eveeka originally received approval in 2021 for treatment in adults and children ages 12 and up.
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Regeneron announced FDA’s approval of its ANGPTL3 antibody Evkeeza for treatment of homozygous familial hypercholesterolemia (HoFH) in children ages 1 to less than 5-years-old. Evkeeza was initially approved in 2021 for use in adults and children aged 12-and-up, and received approval again in 2023 for use in children aged 5 to 11-years-old. Each of Evkeeza’s approvals were reviewed under Priority Review, a process which is reserved for medications representing potential improvements in either efficiency or safety of treating serious diseases.
“The approval of Evkeeza for children as young as 1 year of age addresses a critical unmet need for those with homozygous familial hypercholesterolemia, a life-threatening condition that causes extraordinarily high LDL-C levels from birth,” said Katherine Wilemon, founder and CEO of the Family Heart Foundation. “Families and their medical teams will now have an effective treatment option for these very young children who are at risk of serious complications from diseased arteries and calcified valves without timely and sufficient LDL-C lowering. This development underscores the importance of, and urgency needed in, identifying children with FH through pediatric screenings in accordance with guidelines.”
The new indication of Evkeeza in children ages 1 to less than 5-years-old was supported through safety data and clinical efficacy of six children diagnosed with HoFH, including pharmacokinetic data from four of the children involved in the U.S. expanded access program or ex-U.S. compassionate use program for Evkeeza.1 Within these programs no new safety concerns were identified but the most commonly reported adverse reactions were recording including nasopharyngitis, influenza-like illness, dizziness, rhinorrhea, nausea, and fatigue.
Evkeeza (Evinacumab) is a monoclonal antibody binding and blocking the function of angiopoietin-like 3 (ANGPTL3) protein inhibiting lipoprotein lipase (LPL) and endothelial lipase (EL) while regulating circulating lipids, including LDL-C.1 Evkeeza is the first and only FDA-approved ANGPTL3 inhibitor, indicated for homozygous familial hypercholesterolemia (HoFH). Evkeeza’s success validated ANGPTL3 as a therapeutic target and created opportunities for further innovation for ANGPTL3 therapies, however, Evinacumab’s high cost, as an injectable biologic, presents obstacles for broader adoption. As a results of its high price point, the market is seeing rising interest in alternative modalities, such as antisense oligonucleotides (ASOs) and siRNA therapies.1
“Evkeeza is a testament to the power of Regeneron’s science and proprietary technologies in developing first-in-class, lifechanging medicines that become standard-of-care,” said George D. Yancopoulos, M.D., Ph.D., Board co-chair, president and chief scientific officer at Regeneron. “This label extension adds to our growing commitment to the rare disease space, which includes diverse clinical development programs in neuromuscular and genetic diseases such as myasthenia gravis, otoferlin hearing loss, and fibrodysplasia ossificans progressive, for which we’ve shared data.”
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