Avatrombopag for the treatment of immune thrombocytopenia

Thrombopoietin-receptor agonists (TPO-RAs) are the only American Society of Hematology (ASH) guideline-advocated, second-line treatment for immune thrombocytopenia (ITP) that have been validated by randomized, controlled trials with a placebo comparator. Avatrombopag is a new candidate in this class that has been investigated as a treatment option for the treatment of ITP. Areas covered: In this Drug Profile, we provide a review of the clinical data of avatrombopag, which was approved in May 2018 by the United States Food and Drug Administration (FDA) for the treatment of thrombocytopenia in patients with chronic liver disease undergoing an invasive procedure, and an opinion of its potential place in the current evidence-based ITP treatment landscape. Expert commentary: Avatrombopag induces doubling of platelet counts, increasing them to above 50 X 10⁹/L, and prevents the need for platelet transfusions while minimizing the need for rescue medications. Treatment-emergent adverse events (TEAEs) are comparable to placebo. Oral delivery, a 5-day dosing schedule and good tolerability (<1% discontinuation rate) with no clinically significant hepatoxicity make it a promising entrant as a potential second-line treatment for ITP. Further, data from a phase 3 study in patients with ITP supports its utility in the treatment of patients with ITP.