Health Canada has extended the approval of Evkeeza (evinacumab) to include children as young as 6 months old with homozygous familial hypercholesterolemia (HoFH), marking a significant expansion in treatment options for this devastating rare genetic disorder. The approval represents a critical advancement for pediatric patients facing one of the most severe forms of inherited hypercholesterolemia.
Addressing a Devastating Rare Disease
HoFH is a devastating form of inherited hypercholesterolemia that affects 1 in 300,000 people globally and 1 in 275,000 in the French Canadian population. The condition occurs when two copies of familial hypercholesterolemia-causing genes are inherited, one from each parent, resulting in dangerously high levels of LDL cholesterol exceeding 10 mmol/L. Patients with HoFH face significant risk for premature atherosclerotic disease and cardiac events at an early age, making effective treatment crucial from infancy.
Mechanism of Action and Administration
Evinacumab, the active substance in Evkeeza, operates through a novel mechanism by attaching to the ANGPTL3 protein in the body and blocking its effects. ANGPTL3 plays a key role in controlling cholesterol levels, and blocking its effect reduces cholesterol levels in the blood. The medicine is delivered via monthly infusions administered every four weeks and is used in combination with a low-fat diet and other cholesterol-lowering medications.
Global Availability and Access
Evkeeza is currently reimbursed and commercially available for appropriate HoFH patients in Canada through private drug plans and the public drug program in Quebec. The therapy has also gained approval and commercial availability in the UK, US, Italy, Japan, the Netherlands, Spain, and Luxembourg. Additionally, early access programs have been established in 13 additional countries, including Austria and France, expanding patient access to this critical treatment.
Safety Profile and Considerations
The therapy's safety profile includes several documented side effects, including abdominal pain, back pain, constipation, decreased energy, dizziness, flu symptoms, injection site itchiness, nausea, pain in legs or arms, runny nose, and sore throat or sinus infection. For patients aged 5-11 years old, fatigue has been specifically noted as a side effect. Healthcare professionals must carefully evaluate patients for allergic reactions, including potential anaphylaxis, and consider pregnancy and breastfeeding status before treatment initiation.
Development and Commercialization Partnership
Regeneron Pharmaceuticals discovered and developed Evkeeza, commercializing the product for HoFH in the US under the generic name evinacumab-dgnb. Ultragenyx Pharmaceutical Inc. holds responsibility for development and commercialization efforts in countries outside the United States through a collaboration agreement with Regeneron. This partnership structure enables global access to the therapy while leveraging each company's regional expertise and capabilities.
