Regulatory Decision Summary for Ultomiris

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:


Medicinal Ingredient(s):


Control Number:


Brand/Product Name:


Therapeutic Area:


Type of Submission:

Supplement to a New Drug Submission

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 SNDS was to seek market authorization for Ultomiris (ravulizumab for injection) for the treatment of adult patients with anti-acetylcholine receptor antibody-positive generalized Myasthenia Gravis (gMG).

Ultomiris has been authorized in Canada for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) since August 2019, and for the treatment of adults with atypical hemolytic uremic syndrome (aHUS) since November 2022.

The sponsor consented to information sharing between Health Canada and health technology assessment organizations as part of an aligned review pathway.

Why was the decision issued?

Generalized myasthenia gravis (gMG) is a rare, chronic, debilitating, acquired disorder of neuromuscular transmission that results from the production of pathogenic autoantibodies that bind to components of the neuromuscular junction (e.g., acetylcholine receptor [AChR]), which results in damage and impairment of neuromuscular transmission and weakness of voluntary muscles.

The benefits of Ultomiris were demonstrated by a phase 3, multicentre, randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of ravulizumab in subjects with moderate-to-severe anti-AChR-positive gMG. Subjects receiving ravulizumab achieved a favourable change in patient-reported signs and symptoms of gMG and in physician-reported assessment on the impact of muscle weakness on disease severity. The proportion of subjects achieving a suggestive clinically meaningful benefit in patient-reported signs and symptoms of gMG and physician-reported assessment on the impact of muscle weakness on disease severity, was larger with ravulizumab compared to placebo.

Based on the data submitted, the safety profile of Ultomiris in adults with AChR-positive gMG is generally consistent with that previously established for subjects with PNH and aHUS. Identified risks are considered sufficiently mitigated by product labelling and routine pharmacovigilance activities.

The incidence of adverse events (AEs) was generally consistent when comparing ravulizumab relative to placebo, with few serious AEs, AEs of special interest, and discontinuations due to AEs reported. Treatment-emergent AEs were generally of mild-to-moderate intensity. The most frequently reported treatment-emergent AEs was dizziness and diabetes mellitus; however, these incidences were relatively low. Serious AEs were generally reported as single incidences with no observable trend suggestive of a greater risk of serious AEs in subjects receiving ravulizumab relative to placebo.

Overall, based on the data evaluated as part of this submission, the BRDD considers the benefit-risk profile of Ultomiris to be favourable for the treatment of adults with anti-acetylcholine receptor (AChR) antibody-positive generalized Myasthenia Gravis (gMG).

An updated Risk Management Plan (RMP) for Ultomiris was reviewed by Health Canada and considered acceptable.

Following review and requested revisions, the final labelling and Product Monograph were considered to be acceptable.

For further details about Ultomiris, please refer to the Product Monograph, approved by Health Canada and available through the Drug Product Database.

Date of Decision:



Alexion Pharma Canada

Drug Identification Number(s) Issued:


Prescription Status:

Schedule D drug

Date Filed: