publication date: May. 25, 2018
Drugs and Targets
FDA approves Doptelet for thrombocytopenia in chronic liver disease
The FDA has approved Doptelet (avatrombopag) for thrombocytopenia in adults with chronic liver disease scheduled to undergo a procedure.
The sponsor is AkaRx Inc.
Approval was based on two international, identically designed, randomized, double-blind, placebo-controlled trials, ADAPT-1 and ADAPT-2. Patients (n=430) with chronic liver disease and thrombocytopenia received either avatrombopag (n=274) or placebo (n=156) daily for 5 days prior to a scheduled procedure, and had at least 1 post-dose safety assessment.
Patients were randomized (2:1) to avatrombopag or placebo and stratified according to hepatocellular cancer status and bleeding risk associated with the elective procedure (low, moderate, or high).
Patients randomized to avatrombopag received differential dosing based on mean platelet count at entry. Patients with a baseline platelet count less than 40,000/uL (low baseline platelet cohort) received avatrombopag 60 mg orally once daily for 5 consecutive days.
Those with a baseline platelet count of 40,000 to less than 50,000/uL (high baseline platelet cohort) received avatrombopag 40 mg orally once daily for 5 consecutive days. Eligible patients were scheduled to undergo their procedure 5 to 8 days after the last dose of study drug.
The primary efficacy endpoint was the proportion of patients, or responders, who did not require a platelet transfusion or any rescue procedure for bleeding after randomization and up to 7 days following an elective procedure.
In the low baseline platelet cohort, 66 percent and 69 percent of patients treated with avatrombopag responded in the ADAPT-1 and ADAPT-2 trials, respectively. For those receiving placebo, 23 percent (treatment difference 43%; 95% CI: 27, 58; p<0.0001) and 35% (treatment difference 34%; 95% … Continue reading FDA approves Doptelet for thrombocytopenia in chronic liver disease
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