Regulatory Decision Summary for Evkeeza
The Regulatory Decision Summary explains Health Canada’s decision for the product seeking market authorization. The Regulatory Decision Summary includes the purpose of the submission and the reason for the decision.
Lipid modifying agents
Type of Submission:
New Drug Submission (New Active Substance) - Priority Review
Authorized; issued a Notice of Compliance in accordance with the Food and Drug Regulations
What was the purpose of this submission?
The purpose of this new drug submission was to seek authorization for the use of Evkeeza. After evaluation of the submitted data package, Health Canada authorized Evkeeza for the following indication: Evkeeza (evinacumab for injection) is indicated as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C) lowering therapies for the treatment of adult and pediatric patients aged 5 years and older with homozygous familial hypercholesterolemia (HoFH). The effects of Evkeeza on cardiovascular morbidity and mortality have not been determined.
Why was the decision issued?
Homozygous familial hypercholesterolemia (HoFH) is a rare autosomal recessive genetic disorder that leads to abnormally high low-density lipoprotein cholesterol (LDL-C) in the blood. This leads to atherosclerosis at a younger age than usual and then to cardiovascular disease and death.
Health Canada granted the application for Evkeeza priority review status. Authorization was based primarily on one well-designed, placebo-controlled, multicentre, Phase 3 clinical study. Two other well-designed, multicentre, open-label Phase 3 studies were provided as supportive evidence.
In all three studies, patients were treated with the recommended dose of Evkeeza (15 mg evinacumab per kilogram body weight administered by intravenous [IV] infusion over 60 minutes every four weeks) in addition to any combination of LDL-C lowering therapies (e.g., statins, ezetimibe, proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors, lomitapide, and/or lipoprotein apheresis). The primary efficacy endpoint was percent change in calculated LDL-C level in the blood from baseline to week 24. In adults, adolescents and pediatric patients aged 5 and older treated with Evkeeza, LDL-C levels were lowered by approximately 48-55%.
The most frequently reported adverse events in Evkeeza-treated patients included allergic reactions, infusion-related reactions, temporary changes in blood pressure and heart rate, cold and flu-like symptoms, nausea, weakness, and dizziness. The events reported in pediatric patients were consistent with adults and adolescent patients, with the addition of fatigue.
Risks of Evkeeza treatment have been communicated in the recommended product monograph and will continue to be monitored post-market as outlined in the Risk Management Plan, with routine and non-routine pharmacovigilance activities, including two post-authorization safety studies.
Overall, the benefits of Evkeeza treatment outweigh the risks when it is used according to its recommended conditions of use.
Date of Decision:
Manufacturer / Sponsor:
Drug Identification Number(s) Issued:
Available by prescription only