Regulatory Decision Summary for ALPROLIX
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?
Alprolix (recombinant factor IX) is currently approved for the treatment of previously treated adults and adolescents ≥12 years with idiopathic Factor IX deficiency (Hemophilia B).
This SNDS seeks to extend the currently approved indication to include use of Alprolix in children <12 years with hemophilia B and for its use to reduce bleeding risk during and after surgery for sufferers of all ages.
Why was the decision issued?
The sponsor supplied evidence in the form of a clinical study in 30 children to demonstrate efficacy and safety both for prophylaxis (regular dosing) and for treatment of bleeding episodes. There were no serious side effects recorded in this group of previously treated children with congenital factor IX (FIX) deficiency that had been previously tolerant of FIX replacement therapy.
Alprolix was shown to have a prolonged duration of effect as compared with the previously used product reducing the frequency of dosing required to maintain therapeutic effect.
The residual uncertainties include safety for use in previously untreated children and the possibility of uncommon side effects undetected because of the small numbers studied and the rarity of the condition.
Evidence from both adult and pediatric studies, was also supplied for the safety and efficacy of Alprolix in the management of bleeding at the time of both major and minor surgery.
Health Canada concludes that the benefit already described for Alprolix in adult sufferers with hemophilia B extends to children with the condition and that Alprolix may be used to reduce bleeding in all age groups in the event of surgery.
Decision issued
Approved; issued Notice of Compliance in accordance with the Food and Drug Regulations.