Regulatory Decision Summary for Evkeeza
Review decision
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.
Product type:
Contact:
Medicinal Ingredient(s):
Evinacumab
Control Number:
291743
Brand/Product Name:
Evkeeza
Therapeutic Area:
C10 Lipid Modifying Agents
Type of Submission:
Supplement to a New Drug Submission - Priority Review
Decision Issued:
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 Supplemental New Drug Submission is to expand the authorization for Evkeeza (evinacumab), as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C) lowering therapies, for the treatment of pediatric patients aged 6 months to 5 years with homozygous familial hypercholesterolemia (HoFH).
Why was the decision issued?
This submission proposes extending the indication for Evkeeza to pediatric HoFH patients 6 months to less than 5 years of age. Conducting clinical trials in this rare population is not feasible. Thus, the primary evidence relies on model-based extrapolation informed by population pharmacokinetic/pharmacodynamic (PK/PD) modelling and assumptions on disease similarity and biological development. The International Council for Harmonisation, ICH E11A (Guideline for Pediatric Extrapolation) emphasizes that for extrapolation to be appropriate, there must be sufficient evidence that: the disease course and pathophysiology are sufficiently similar between the source (subjects older than 5 years of age) and target populations (subjects 6 months to 5 years of age); and, the response to treatment is expected to be similar, based on biology, pharmacology, or existing data.
Supportive data come from five patients treated under compassionate use programs, all showing substantial LDL-C reductions. Simulations and observational data confirm that 15 mg/kg IV Q4W achieves consistent LDL-C lowering in children 6 months to < 5 years of age, in a manner similar to what was observed in older children, adolescents, and adults, with no need for dose adjustment in this patient population. The totality of evidence, including disease pathophysiology, PK/PD modelling, and clinical observations, justifies the extension of use of Evkeeza in children aged 6 months to less than 5 years.
The Marketed Health Products Directorate (MHPD) has determined the Evkeeza Global Risk Management Plan (RMP) version 2.0 and Canadian Regional Annex to the RMP to be acceptable.
The labelling information submitted met all applicable guidance and regulations.
For further details about Evkeeza, please refer to the Product Monograph, approved by Health Canada and available through the Drug Product Database.
Date of Decision:
2025-07-04
Manufacturer/Sponsor:
Drug Identification Number(s) Issued:
N/A
Prescription Status:
Available by prescription only
Date Filed:
2024-11-04
Related Drug Products
| Product name | DIN | Company name | Active ingredient(s) & strength |
|---|---|---|---|
| EVKEEZA | 02541769 | ULTRAGENYX PHARMACEUTICAL INC | EVINACUMAB 150 MG / ML |