Regulatory Decision Summary for Ultomiris
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.
Type of Submission:
Supplement to a New Drug Submission
Authorized; issued a Notice of Compliance in accordance with the Food and Drug Regulations
What was the purpose of this submission?
After evaluation of the submitted data package, Health Canada authorized Ultomiris (ravulizumab) for the treatment of adult patients with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody-positive. Ultomiris has been authorized in Canada since 2019. It is currently authorized for the treatment of adult and pediatric paroxysmal nocturnal hemoglobinuria, as well as for the treatment of adult and pediatric atypical hemolytic uremic syndrome and for generalized myasthenia gravis in adults.
Why was the decision issued?
Authorization was based on one phase 3, single-arm, open-label, multicentre study in adult patients with NMOSD. A total of 58 patients with NMOSD were exposed to Ultomiris, with a median (min, max) study duration of 73.50 (13.7, 117.7) weeks. The assessment of efficacy was based on a comparison with an external historical placebo group. This approach was considered acceptable. The primary efficacy endpoint was the time to first adjudicated on-trial relapse and relapse risk reduction. The primary endpoint was met, all patients on Ultomiris were relapse-free at the end of the Primary Treatment Period (versus 63.2% of placebo-treated patients).
The most common adverse reactions (ADRs) reported in at least 10% of patients were: COVID-19 (24.1%), headache (24.1%), back pain (12.1%), arthralgia (10.3%), and urinary tract infection (10.3%). These findings are consistent with the previously authorized indications for Ultomiris. The most notable risk for Ultomiris is an increased susceptibility to meningococcal infections. This is labelled in a Serious Warnings and Precautions box in the Canadian Product Monograph. Overall, the benefit-risk profile of Ultomiris used in the treatment of adult patients with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody-positive was deemed favorable.
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Ultomiris is both a Prescription and a Schedule D drug