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 trial; 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 responded or did not require a platelet transfusion or any rescue procedure for bleeding after randomization and up to 7 days following an elective procedure.
Among patients with the lowest baseline platelet counts, 66 percent and 69 percent of patients treated with avatrombopag responded in the ADAPT-1 and ADAPT-2 trials, respectively, compared with 23 percent and 35 percent of patients who received placebo. Among those with the highest baseline platelet cohorts, 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 (reported in ≥3% of patients) were pyrexia, abdominal pain, nausea, headache, fatigue, and peripheral edema.
Source: U.S. FDA news release, May 21, 2018.