Health Canada approves Doptelet (avatrombopag) to treat chronic immune thrombocytopenia

Avatrombopag is an oral thrombopoietin (TPO) receptor agonist that stimulates the proliferation and differentiation of megakaryocytes from bone marrow progenitor cells, resulting in increased production of platelets. It does not compete with TPO for binding to the TPO receptor.

Health Canada based these approvals on results from several clinical trials. In a phase III study of adult patients with immune thrombocytopenia, administration of avatrombopag resulted in a platelet count of at least 50,000 per L at day eight of therapy in most patients. Avatrombopag was superior to placebo in maintaining platelet counts in the target range during the six-month treatment period. Among patients with thrombocytopenia and CLD, findings from the phase III ADAPT-1 and ADAPT-2 trials demonstrated that the proportion of patients not requiring a platelet transfusion or any rescue procedure for bleeding was significantly higher in the two avatrombopag-treated groups.