The Food and Drug Administration (FDA) has approved Doptelet (avatrombopag; Dova Pharma) for the treatment of thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo a procedure.

Doptelet is an orally bioavailable, small molecule thrombopoietin receptor agonist that stimulates proliferation and differentiation of megakaryocytes from bone marrow progenitor cells resulting in an increased population of platelets. “Patients with chronic liver disease who have low platelet counts and require a procedure are at increased risk of bleeding,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “Doptelet was demonstrated to safely increase the platelet count. This drug may decrease or eliminate the need for platelet transfusions, which are associated with risk of infection and other adverse reactions.”

The approval of Doptelet was based on results from the ADAPT-1 (N=231) and ADAPT-2 trials (N=204), 2 identically-designed multicenter, randomized, double-blind, placebo-controlled studies. Study results showed that patients in the Doptelet treatment groups had a greater proportion of responders (defined as patients who did not require a platelet transfusion or any rescue procedure for bleeding after randomization and up to 7 days following a scheduled procedure) than the corresponding placebo treatment groups (ADAPT-1: low baseline platelet count cohort [60mg dose]: 66% vs 23%; P<.0001; high baseline platelet count cohort [40mg dose]: 88% vs 38%; P<.0001; ADAPT-2: low baseline platelet cohort [60mg dose]: 69% vs 35%; P=.0006; high baseline platelet cohort [40mg dose]: 88% vs 33%; P<.0001).

The most common adverse reactions associated with Doptelet in clinical trials were pyrexia, abdominal pain, nausea, headache, fatigue, and peripheral edema. The use of Doptelet may increase the risk of thrombotic and thromboembolic complicatons in patients with chronic liver disease. This potential increased thrombotic risk should be considered when administering Doptelet to patients with known risk factors for thromboembolism, including genetic prothrombotic conditions.

Doptelet is supplied as 20mg tablets and is expected to be available sometime in June 2018.

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