FDA Approves New Drug to Treat Thrombocytopenia in Chronic Liver Disease

The FDA has approved Doptelet (avatrombopag; AkaRx Inc.), a drug used for the treatment of thrombocytopenia in adult patients with chronic liver disease who have low blood platelets and are scheduled to undergo a medical or dental procedure. This is the first drug approved by the FDA for this use.

Thrombocytopenia is a condition in which there is a lower-than-normal number of circulating platelets in the blood, which can result in serious or life-threatening bleeding, especially during invasive procedures. Thrombocytopenia is a common hematological abnormality encountered in patients with chronic liver disease. Patients with significant thrombocytopenia typically receive platelet transfusions immediately prior to a procedure to increase their platelet count.

“Patients with chronic liver disease who have low platelet counts and require a procedure are at increased risk of bleeding,” said Richard Pazdur, MD, 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 safety and efficacy of Doptelet was studied in 2 trials involving 435 patients with chronic liver disease and severe thrombocytopenia who were scheduled to undergo a procedure that would typically require platelet transfusion. The trial results showed that a higher proportion of patients who received Doptelet had increased platelet counts and did not require platelet transfusion or any rescue therapy on the day of the procedure and up to 7 days following the procedure compared with those who were treated with placebo.

Doptelet dosing should begin 10 to 13 days prior to a scheduled procedure. The recommended daily dose of Doptelet is based on the patient’s platelet count prior to the scheduled procedure. Doptelet is taken orally, once daily for 5 consecutive days. Patients should undergo their procedure 5 to 8 days after the last dose.

Additional information about Doptelet can be found here.