Ultragenyx Pharmaceuticals has announced the extension of approval by Health Canada for Evkeeza (evinacumab) as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C) lowering treatments to include children aged six months and above with homozygous familial hypercholesterolemia (HoFH).
This decision follows the initial approval granted in September 2023, which covered patients aged five and above.
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The drug was discovered and developed by Regeneron Pharmaceuticals, which commercialises it in the US.
Ultragenyx is responsible for its development as well as commercialisation in countries outside of the US.
The pharmacokinetics and efficacy data of Evkeeza were derived from model-based extrapolation analysis.
These analyses predict that younger paediatric patients – from six months to under five years – will experience a similar or greater reduction in LDL-C levels after 24 weeks when administered a dose of 15mg/kg every four weeks, compared to older children and adults.
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By GlobalDataSupportive data from five young patients, aged one to four years, showed meaningful reductions in LDL-C levels, consistent with clinical study results seen in older age groups.
Ultragenyx Canada vice-president and general manager Monty Keast stated: “HoFH is a severe, life-threatening condition that, if left untreated, can lead to heart disease and death as early as childhood. This makes diagnosing it and reducing the resulting high LDL-C levels an urgent need.
“We are proud to be able to provide children as young as six months old living with HoFH a medicine that could reduce LDL-C levels and will work collaboratively with healthcare providers and payers across the country to ensure it is accessible for families.”
Evkeeza’s active substance evinacumab works by targeting angiopoietin-like protein 3 (ANGPTL3) that regulates cholesterol levels. In January 2025, the European Commission (EC) expanded the approval of Evkeeza to include children aged six months to under five years diagnosed with HoFH.
