FDA Approves Avatrombopag for Thrombocytopenia

The U.S. Food and Drug Administration (FDA) has approved avatrombopag for the treatment of thrombocytopenia in adults with chronic liver disease who are scheduled to undergo a medical or dental procedure. This is the first drug approved for this indication.

The FDA’s decision was based on data from two randomized, double-blind, placebo-controlled trials (ADAPT-1 and ADAPT-2). A total of 430 patients were enrolled in the trials: 274 received avatrombopag and 156 received placebo daily for five days prior to a scheduled procedure.

The primary efficacy endpoint in each trial was the proportion of patients who did not require a platelet transfusion or any rescue procedure for bleeding after randomization and up to seven days following an elective procedure.

Among patients with the lowest baseline platelet counts, 66 percent and 69 percent of avatrombopag-treated patients responded in the ADAPT-1 and ADAPT-2 trials, respectively, compared with 23 percent and 35 percent of placebo-treated patients. Among those with the highest baseline platelet counts, 88 percent of avatrombopag-treated patients in both trials responded to treatment, compared with 38 percent and 33 percent of placebo-treated patients in ADAPT-1 and ADAPT-2.

The most common adverse events (AEs; reported in ≥3% of patients) were pyrexia, abdominal pain, nausea, headache, fatigue, and peripheral edema.

Source: U.S. FDA news release, May 21, 2018.