Regulatory Decision Summary for Zonovate
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.
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What was the purpose of this submission?
Zonovate (Antihemophilic Factor (Recombinant, B-Domain Truncated)) is a purified coagulation Factor VIII (FVIII) obtained by recombinant DNA technology, for the treatment and prevention of bleeding episodes in patients with hemophilia A.
The efficacy information for Zonovate has been updated in severe hemophilia A patients with less than 1% normal activity levels of FVIII, following a completed extension Study 3568 in previously treated patients (PTPs) and an ongoing Study 3809 in previously untreated patients (PUPs).
Why was the decision issued?
Study 3568 provided long-term exposure data in children and adults for preventive treatment, as well as for on-demand treatment and for patients requiring minor or major surgery. Study 3809 enrolled pediatric patients (age range 0-42 months) who have never been previously treated with a Factor VIII (FVIII) product, in order to evaluate the incidence of antibodies against FVIII (inhibitors), a common complication in previously untreated patients (PUPs) with any FVIII product.
Overall, Zonovate was shown to be efficacious and well tolerated in both previously treated patients (PTPs) and PUPs. The common risks related with the use of Zonovate included injection site reactions and incorrect dose administered. In PUPs, the incidence of inhibitors with Zonovate was as expected for this age group. PUPs with inhibitors were also at risk of infections related to the catheter site or device during immune tolerance induction therapy. In PTPs, only rare cases of inhibitor development were reported during post-market experience.
The benefit in the use of Zonovate is considered favorable over the risks as described in the Product Monograph. Risk mitigation had been addressed in the proposed Product Monograph, including precautionary statements for patients developing inhibitors and recommendations for immune tolerance treatment.
Decision issued
Approved; issued a Notice of Compliance in accordance with the Food and Drug Regulations.