FDA approves Doptelet for thrombocytopenia.- Dova Pharmaceuticals.

The FDA has approved Doptelet (avatrombopag) tablets, from Dova Pharmaceuticals, to treat low blood platelet count (thrombocytopenia) in adults with chronic liver disease who are scheduled to undergo a medical or dental procedure. This is the first drug approved by the FDA for this use.

The safety and efficacy of Doptelet was studied in two trials (ADAPT-1 and ADAPT-2) involving 435 patients with chronic liver disease and severe thrombocytopenia who were scheduled to undergo a procedure that would typically require platelet transfusion. The trials investigated two dose levels of Doptelet administered orally over five days as compared to placebo (no treatment). The trial results showed that for both dose levels of Doptelet, a higher proportion of patients had increased platelet counts and did not require platelet transfusion or any rescue therapy on the day of the procedure and up to seven days following the procedure as compared to those treated with placebo.

The most common side effects reported by clinical trial participants who received Doptelet were fever, stomach (abdominal) pain, nausea, headache, fatigue and swelling in the hands or feet (edema). People with chronic liver disease and people with certain blood clotting conditions may have an increased risk of developing blood clots when taking Doptelet.

Comment: Avatrombopag is a second generation orally administered TPO-RA which is intended to address the limitations of other existing treatments for thrombocytopenia. Avatrombopag is initially being studied for its potential for the acute treatment of thrombocytopenia in patients with CLD scheduled to undergo a procedure. Dova is also planning to explore the potential use of avatrombopag in a broader population of patients with thrombocytopenia, including in patients undergoing surgical procedures associated with a high risk of bleeding, and patients who develop thrombocytopenia after receiving chemotherapy.