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:

Drug

Medicinal ingredient(s):

Ravulizumab

Therapeutic area:

Immunosuppressants

Type of submission:

New Drug Submission

Control number:

217955
What was the purpose of this submission?

 

The purpose of this submission is to seek marketing authorisation for Ultomiris in Canada for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH).

 

Why was the decision issued?

 

The benefits of Ultomiris (ravulizumab) in the target patient population were demonstrated in two pivotal clinical trials comparing Ultomiris and Soliris.

Study ALXN1210-PNH-301 was conducted in 246 patients with paroxysmal nocturnal hemoglobinuria (PNH) who were naïve to complement inhibitor treatment. The co-primary endpoints were transfusion avoidance, and reduction of hemolysis as directly measured by normalization of serum lactate dehydrogenase (LDH) levels. Key secondary endpoints included the percent change from baseline in LDH levels, change in quality of life, the proportion of patients with breakthrough hemolysis, and proportion of patients with stabilized hemoglobin. Both co-primary endpoints and all 4 key secondary endpoints were met.

Study ALXN1210-PNH-302 was conducted in 195 patients with PNH who were clinically stable after having been treated with Soliris for at least the previous 6 months. The primary endpoint was hemolysis as measured by LDH percent change from baseline. Secondary endpoints included the proportion of patients that experienced breakthrough hemolysis, quality-of-life, transfusion avoidance, and proportion of patients with stabilized hemoglobin. The primary endpoint and all 4 key secondary endpoints were met.

In both trials, Ultomiris was shown to be statistically non-inferior compared to Soliris and the results were clinically meaningful.

A total number of 261 patients with PNH received Ultomiris, representing cumulatively 177 patient-years in clinical trials. The most frequent adverse drug reaction in clinical trials was headache. The most serious adverse reactions in patients in clinical trials were meningococcal infection and meningococcal sepsis. Adequate risk minimization measures were implemented through the Product Monograph and the RMP. Ultomiris will be available in Canada a through a controlled distribution programme.

Overall, based on the clinical data provided and with the risk minimization measures implemented, the benefits of Ultomiris outweigh its risks in the target patient population.

For more information on Health Canadas decision, please view the Summary Basis of Decision.

 

Decision issued

Approved; issued a Notice of Compliance in accordance with the Food and Drug Regulations.