Avatrombopag (Doptelet ®) received its first global approval in the US; this was reviewed in detail in the First Global Approval report in Adis’ journal Drugs[1], based on the development milestones tracked in AdisInsight[2].

In May 2018 avatrombopag, developed by Dova Pharmaceuticals, was approved by the US FDA for use in the treatment of thrombocytopenia in adult patients with chronic liver disease (CLD) who are scheduled to undergo a medical or dental procedure; this is the first drug approved by the FDA for this purpose.[3] Avatrombopag is a second generation, orally bioavailable, small molecule thrombopoietin receptor agonist that increases platelet production by mimicking the effects that thrombopoietin has on megakaryocytes in bone marrow progenitor cells. [1] [4]

Thrombocytopenia is a common symptom in patients with CLD; these patients often require invasive procedures resulting in the need for platelet transfusions to prevent bleeding. The transfusions are associated with their own set of complications, including risk of infections.[4]

Avatrombopag was approved under Priority Review by the FDA and supported by evidence from the ADAPT-1[5] and ADAPT-2[6] trails in a total of 435 patients with chronic liver disease and severe thrombocytopenia who were scheduled to undergo a procedure.

Professor Norah Terrault, from the University of California San Francisco, Division of Gastroenterology, and Principal Investigator for the Phase 3 pivotal avatrombopag trials said, “Given the need for patients with CLD to routinely undergo multiple, invasive procedures, the availability of an oral agent that can lead to a measured increase in platelets, to minimize the need for platelet transfusions and risk of bleeding, will facilitate the clinical management of these patients.” [4]

The prescribing information for avatrombopag cautions against thrombotic/thromboembolic complications, which is not surprising given its mechanism of action.[7]

Avatrombopag is also under regulatory review in Europe for the same indication.[1]

For further information related to the first approval of avatrombopag please visit Drugs[1],  or to learn more about the overall development of the drug across all indications visit AdisInsight. [2]

Image credit:  Christian Holzinger -Unsplash

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