Pune, India | October 03, 2025
The U.S. Food and Drug Administration (FDA) has approved Evkeeza® (evinacumab‑dgnb) for children as young as one year diagnosed with homozygous familial hypercholesterolemia (HoFH), a rare genetic disorder leading to extremely high LDL cholesterol from infancy and significantly increasing the likelihood of early cardiovascular disease and stroke. This approval marks an important step forward in managing one of the most aggressive lipid disorders, which, without treatment, can dramatically shorten life expectancy.
Previously, the therapy was approved strictly for adults and adolescents aged twelve and older, later extending to children between five and eleven years. Now, with the recent decision, children as young as one year gained access to this life‑saving therapy, giving families vital opportunities to begin treatment earlier and potentially prevent severe complications in the future. The extension solidifies Evkeeza’s role as a cornerstone in HoFH treatment while expanding the scope of pediatric care.
The FDA based its decision on clinical data from six young patients, including detailed pharmacokinetic studies on four of them. Importantly, during the evaluation process, no previously unknown safety risks were identified. Considering that HoFH patients commonly exhibit LDL cholesterol levels above 400 mg/dL, which fuel rapid atherosclerosis and early cardiovascular events even during adolescence, timely treatment remains critical. Although fewer than 1,300 people in the United States suffer from the condition, early diagnosis and medical intervention significantly impact patient outcomes.
Evkeeza operates by inhibiting a protein called angiopoietin‑like 3 (ANGPTL3), which plays a crucial role in lipid metabolism. By blocking this protein, the drug successfully reduces LDL cholesterol and other harmful lipid markers circulating in the blood. Previous studies among adults and older children revealed that Evkeeza, when combined with standard cholesterol‑lowering therapies, can cut LDL cholesterol levels nearly in half.
In addition, patients experienced consistent reductions in apolipoprotein B and non‑HDL cholesterol, further proving the medication’s effectiveness. Used alongside diet adjustments, regular physical activity, and other therapies, Evkeeza strengthens clinicians’ ability to fight the damaging effects of this rare disorder.
Safety continues to guide treatment decisions, particularly with very young patients who may face unknown long‑term risks. Reported side effects include nasopharyngitis, flu‑like symptoms, dizziness, runny nose, nausea, and fatigue. Nevertheless, in children studied under the most recent approval data, no unexpected adverse reactions emerged.
Even with encouraging findings, physicians remain cautious because this approval introduces treatment in an age group where little long‑term information exists. Continued monitoring and further studies remain vital to ensure safety as treatment begins earlier in life.
Despite clinical benefits, treatment accessibility poses practical challenges. As a biologic therapy, Evkeeza involves high costs, making insurance coverage complex for many families. To assist, Regeneron, the drug’s manufacturer, supports patients through its myRARE program, designed to help families troubleshoot insurance barriers while also addressing financial concerns. This effort reflects the broader recognition that medical innovations only achieve their purpose when patients can realistically access them.
The FDA’s decision represents a pivotal advance not only for affected children but for the rare disease community at large. It underscores the importance of precision medicine approaches that target specific genetic pathways previously considered untreatable.
Furthermore, the approval strengthens the case for early genetic screening and comprehensive cholesterol testing in children from high‑risk families. By diagnosing familial hypercholesterolemia earlier, healthcare providers can intervene before irreversible cardiac damage develops.
In addition to broadening the treatment age range, this regulatory win emphasizes the urgent need for sustained research and medical innovation in pediatric rare conditions. Experts believe initiating cholesterol‑lowering therapy in very young children might lower the likelihood of catastrophic cardiac events later in life, including heart attacks and strokes.
Still, specialists must carefully weigh the proven advantages of early treatment against the uncertainties of prolonged use, ensuring clinical best practices constantly evolve with emerging data.
Ultimately, FDA approval of Evkeeza for children as young as one represents a medical milestone in the fight against ultra‑rare and life‑threatening high‑cholesterol disorders. This decision provides families with new hope, empowering clinicians to intervene earlier in a disease notorious for causing devastating consequences. Most importantly, it demonstrates how advanced biotechnology and genetic understanding can profoundly transform care models, enabling patients with otherwise untreatable inherited disorders to envision healthier, longer futures.
