EC Expands Evkeeza® Approval to Children 6 Months+ with HoFH

On January 6, 2025, Ultragenyx Pharmaceutical Inc., a company focusing on innovative treatments for rare genetic disorders, announced that Evkeeza® (evinacumab) has received extended approval from the European Commission. This new approval allows the drug to be used alongside diet and other lipid-lowering treatments for children as young as six months with homozygous familial hypercholesterolemia (HoFH). Evkeeza, an inhibitor of angiopoietin-like 3 (ANGPTL3), is now the first medication in the European Union indicated for treating HoFH in such young patients. HoFH is marked by critically high levels of low-density lipoprotein cholesterol (LDL-C), often leading to early cardiovascular issues.

Albert Wiegman, M.D., Ph.D., a leading pediatrician at Amsterdam University Medical Center, emphasized the significance of this milestone. He noted that young children with HoFH face limited treatment options, often failing to meet their cholesterol management goals, which jeopardizes their future health. According to Dr. Wiegman, evinacumab has shown a substantial reduction in LDL-C levels in adults, adolescents, and children aged five and older, and the recent label expansion highlights its potential in managing LDL-C levels in even younger children.

The European Commission’s decision follows a favorable recommendation from the Committee for Medicinal Products for Human Use issued in November 2024. The effectiveness of Evkeeza in children aged six months to five years with HoFH has been supported by model-based extrapolation analysis. Findings indicate that these young patients could exhibit similar or even greater reductions in LDL-C levels at week 24 with a 15 mg/kg dose every four weeks, compared to adults. Additionally, data from five children aged one to four who received the drug through compassionate use demonstrated clinically significant LDL-C reductions akin to those in older children and adults in previous studies. The safety profile for young patients appears consistent with that of older children, with no new safety concerns identified.

Magdalena Daccord, CEO of the FH Europe Foundation, welcomed the approval and highlighted its potential to transform lives. She underscored the importance of early screening and detection for HoFH, advocating for the availability of effective treatment options for young patients. She expressed hope for widespread access to this life-changing therapy for those who need it promptly.

Previously, Evkeeza was approved in June 2021 for treating adolescents and adults with HoFH. The indication was later extended to children aged five to eleven in December 2023. The treatment is currently available in several countries, including the UK, US, Canada, and Japan, and is accessible through early access programs in additional nations.

Jane Cooper, senior vice president at Ultragenyx, expressed pride in providing the first approved treatment for children as young as six months with HoFH. She emphasized the urgent need to address the high LDL-C levels in these young patients to prevent severe cardiovascular events.

HoFH is a severe genetic condition affecting approximately 1 in 300,000 people worldwide. It results from inheriting two faulty genes, leading to dangerously high LDL-C levels. Patients with HoFH face a heightened risk of early-onset atherosclerotic disease and cardiac events. Evkeeza works by inhibiting the ANGPTL3 protein, which plays a role in cholesterol regulation, thus reducing cholesterol levels in the blood. The medication is administered through monthly infusions.

Evkeeza's discovery and initial development were led by Regeneron Pharmaceuticals, Inc. Ultragenyx manages the drug's development and commercialization outside the United States. The company remains committed to delivering groundbreaking therapies for rare diseases, prioritizing safe and effective treatment options with efficient development processes.