Regulatory Decision Summary for Ultomiris

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.

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.