Regulatory Decision Summary for NiaStase RT
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?
Niastase RT is currently authorized in Canada for the following indications:
In hemophilia A/B patients with inhibitors to FVIII or FIX, respectively, for the treatment of bleeding episodes (including treatment and prevention of those occurring during and after surgery); for the treatment of severe bleeding episodes in Glanzmanns thrombasthenia with clinical refractoriness and/or platelet-specific antibodies, or where platelets are not immediately available; for the prevention of bleeding in surgical interventions or invasive procedures in Glanzmanns thrombasthenia with clinical refractoriness and/ or platelet-specific antibodies, or where platelets are not readily available; in adult patients with acquired hemophilia, for the treatment of bleeding episodes, and for the prevention of bleeding in those undergoing surgery or invasive procedures.
The purpose of this Supplemental New Drug Submission (SNDS) was to seek the marketing authorization for NiaStase RT for an additional indication: in patients with congenital Factor VII deficiency, for the treatment and routine prophylaxis of bleeding episodes, and for the prevention of bleeding in those undergoing surgery or invasive procedures.
Why was the decision issued?
Reported results from a registry that collected data on treatment modalities and outcomes in patients with congenital Factor VII deficiency have shown a good efficacy profile for Niastase RT in the treatment of bleeding episodes and in the prevention of bleeding in those undergoing surgery or invasive procedures. There were no sufficient data to support the indication for the routine prophylaxis of bleeding episodes.
No new safety concerns were identified other than the risk of thrombotic adverse events, which is already known with the product and for which risk management and mitigation strategies are in place.
Given the rarity of Congenital Factor VII deficiency, and the fact that no therapies have been approved for this condition in Canada, it has been determined that, despite the limitations of the data from a registry, the benefit risk profile of Niastase RT is considered favourable in patients with congenital factor VII deficiency for the treatment of bleeding episodes, and for the prevention of bleeding in those undergoing surgery or invasive procedures.
Decision issued
Approved; issued a Notice of Compliance in accordance with the Food and Drug Regulations.