Pune, India | October 03, 2025
The U.S. Food and Drug Administration (FDA) recently expanded the approval for Evkeeza® (evinacumab-dgnb), allowing its use in children as young as one year old. This crucial decision targets children suffering from homozygous familial hypercholesterolemia (HoFH), a rare genetic disorder. HoFH forces dangerously high levels of LDL cholesterol, often called “bad” cholesterol, into the body right from infancy. Furthermore, this severe condition dramatically increases a child’s risk of experiencing an early heart attack or stroke.
Previously, the FDA had only approved Evkeeza for adolescents and adults aged 12 and older, before later extending it to children between the ages of five and 11. Consequently, this new approval now extends this vital treatment option to a much younger patient population. This provides a crucial opportunity for early intervention, which could potentially prevent devastating, irreversible cardiovascular damage later in life. We must prioritize finding treatments for the youngest patients fighting these rare conditions.
The support for this significant expansion comes from clinical data collected from six young children diagnosed with HoFH. Researchers also completed pharmacokinetic studies in four of these young participants. Importantly, the evaluation process did not identify any new safety concerns, reassuring clinicians about its use. Because HoFH represents one of the most severe forms of familial hypercholesterolemia, patients with this condition routinely exhibit LDL cholesterol levels surpassing 400 mg/dL.
This extreme level aggressively accelerates atherosclerosis, causing cardiovascular events sometimes as early as the teenage years. Although the condition is exceptionally rare, affecting only about 1,300 people in the United States, early diagnosis and swift treatment remain absolutely critical.
Evkeeza operates by actively targeting and blocking a specific protein called ANGPTL3, a molecule that significantly regulates lipid metabolism. By effectively inhibiting ANGPTL3, Evkeeza works to lower LDL cholesterol and other harmful lipid particles circulating in the bloodstream. Previous clinical trials involving older children and adults demonstrated the drug’s significant effectiveness.
For instance, adding Evkeeza to patients’ standard cholesterol-lowering therapies reduced their LDL cholesterol levels by almost 50%. Simultaneously, patients also saw significant decreases in other key lipid markers like apolipoprotein B and non-HDL cholesterol. Ultimately, when physicians combine Evkeeza with diet, exercise, and other medications, it provides a powerful new tool to manage this life-threatening genetic disorder.
Patient safety remains the paramount concern, particularly when treating very young patients. Clinicians reported common side effects, including nasopharyngitis (common cold), flu-like symptoms, dizziness, runny nose, nausea, and fatigue. Notably, investigators observed no unexpected adverse events among the youngest group studied in the trials. Nevertheless, long-term safety data are still limited because the treatment is now starting at an earlier age than ever before, requiring continued monitoring.
Furthermore, the high cost and complex access to this biologic treatment present undeniable challenges for families. Therefore, Regeneron developed the myRARE patient support program to help families navigate these insurance and financial hurdles, improving access.
The FDA’s decision represents a major scientific advance, benefiting not only the affected children but also the broader rare disease community. This landmark approval powerfully reflects the capability of precision medicine and genetic targeting in developing treatments for conditions we once considered untreatable.
Moreover, the approval highlights the critical importance of early genetic screening and diagnosis. This encourages healthcare providers to identify familial hypercholesterolemia quickly so affected children can begin treatment before serious complications arise.
In addition to expanding Evkeeza’s age range, this approval sends a strong message promoting continued research and innovation in pediatric rare diseases. The decision effectively opens doors for further studies to explore whether lowering LDL cholesterol at such a young age will reduce the incidence of heart attacks and strokes much later in patients’ lives. Clinicians must carefully and continually balance the clear benefits of early treatment with ongoing patient monitoring to ensure safety and effectiveness over time.
In conclusion, the FDA’s approval of Evkeeza for children as young as one with HoFH truly marks a significant leap forward in treating this ultra-rare and severe condition. This life-saving drug provides a new lifeline to very young patients who face devastating health risks without effective therapy.
Most importantly, this advancement clearly demonstrates how advanced biotechnology and deep genetic insights can transform patient care. This progress offers hope for better outcomes and healthier futures for all children battling inherited high cholesterol disorders.
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