Regulatory Decision Summary for Nplate
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?
Nplate (romiplostim) is currently authorized in Canada for the treatment of adults with chronic immune thrombocytopenia (ITP) who have had an inadequate response or are intolerant to corticosteroids, immunoglobulins and/or splenectomy. The purpose of this submission was to seek expansion of the existing indication to adult patients with newly diagnosed (0-3 months) and persistent (3-12 months) ITP. A Notice of Compliance (NOC) was recommended.
The authorized indication is that Nplate is indicated to increase the platelet levels in adult patients with ITP:
- who are nonsplenectomized and have had an inadequate response or are intolerant to corticosteroids and/or immunoglobulins
- who are splenectomized and have had an inadequate response to splenectomy.
Why was the decision issued?
Authorization was based on the results of a single arm study in adults with immune thrombocytopenia (ITP) who had been diagnosed within 6 months of study enrollment and who had failed one previous ITP treatment. A total of 75 patients received a subcutaneous injection of Nplate at an initial dose of 1 µg/kg followed by 12 months of weekly injections with dosing adjustments based on platelet count. The primary efficacy endpoint was the number of months with a platelet response (≥ 50 x 109/L) during the 12 month treatment period.
The results of the primary efficacy endpoint reported a mean of 9.2 months with a platelet response during the 12 month treatment period. Twenty-four (32%) patients maintained every platelet count ≥ 50 x 109/L for at least 6 months in the absence of Nplate and any medication for ITP.
The most common treatment emergent adverse events reported in at least 10% of patients included nasopharyngitis, arthralgia, headache, and hematoma. Bleeding events were reported in 23 (31%) patients. The safety findings were consistent with the known safety profile for Nplate.
The recommended initial dose for Nplate is 1 µg/kg administered once weekly as a subcutaneous injection. Dosage adjustments are recommended based on platelet count. View the Product Monograph for details.
Decision issued
Authorized; issued a Notice of Compliance in accordance with the Food and Drug Regulations