Regulatory Decision Summary for Hemlibra

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):

Emicizumab

Therapeutic area:

Antihemorrhagic
ATC code: B02BX06

Type of submission:

Supplemental New Drug Submission

Control number:

222209
What was the purpose of this submission?

 

The purpose of this submission was two-fold. The first was to seek an expanded indication for Hemlibra to include hemophilia A patients without inhibitors, who are currently eligible to receive prophylactic Factor VIII (FVIII) replacement therapy. The second was to provide patients and physicians the option of using two new alternative maintenance doses.

 

Why was the decision issued?

 

Authorization was based on a pivotal study including both a randomized and non-randomized component. In the controlled portion, hemophilia A patients without inhibitors to FVIII were randomized 2:2:1 to receive either Hemlibra at the approved 1.5 mg/kg once weekly (QW) maintenance dose (n = 36), Hemlibra at a new 3.0 mg/kg once every 2 weeks (Q2W) maintenance dose (n = 35), or no prophylaxis (n = 18). Hemophilia A patients in the 1.5 mg/kg QW and 3.0 mg/kg Q2W arms had annualized bleed rates of 1.5 and 1.3, respectively, which were lower than the annualized bleed rate of 38.2 reported in hemophilia A patients who received no prophylaxis.

In the non-randomized portion of the study, hemophilia A patients were treated with FVIII prophylaxis in a non-interventional study before switching over to Hemlibra prophylaxis. The study demonstrated that most of the forty-eight patients who received FVIII prophylaxis in the non-interventional study received their intended doses every 2 to 4 days (on average). The study results indicated that Hemlibra prophylaxis resulted in a statistically significant reduction in bleed rate for treated bleeds compared to FVIII prophylaxis. The mean annualized bleed rate for patients on Hemlibra and FVIII prophylaxis were 1.5 and 4.8, respectively.

Additional studies demonstrated that a 3 mg/kg every two week maintenance dose was as effective as the 1.5 mg/kg every week maintenance dose in controlling bleeds in hemophilia patients of all ages, while the 6 mg/kg every four week maintenance dose was effective in adolescents and adults who were twelve years and older weighing forty kilograms or more. No safety concerns were identified outside of events currently listed in the Product Monograph and no thrombotic events were reported in clinical studies of hemophilia A patients without inhibitors to FVIII.

A Notice of Compliance was recommended to expand the indication of Hemlibra for the treatment of hemophilia A patients without inhibitors to FVIII, including the recommendation for two new Hemlibra maintenance doses.

 

Decision issued

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