FDA/CDC

FDA approves second drug for thrombocytopenia in liver disease


 

The Food and Drug Administration has approved lusutrombopag (Mulpleta) for the treatment of thrombocytopenia in patients with chronic liver disease who are scheduled to undergo medical or dental procedures. The drug is expected to be available in the United States in September 2018.

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In May 2018, the FDA approved avatrombopag (Doptelet) as the first treatment for thrombocytopenia in this patient population.

The approval is based on a pair of phase 3, double-blind, placebo-controlled clinical trials – L-PLUS 1 and L-PLUS 2 (NCT02389621) – that included 312 patients who have chronic liver disease with severe thrombocytopenia. In L-PLUS 1, the primary endpoint was the percentage of patients who didn’t need platelet transfusions before the primary procedure was performed; this was met by 78% in the lusutrombopag group versus just 13% in the control group. In L-PLUS 2, the primary endpoint was the percentage of patients who didn’t need platelet transfusions before the procedure and didn’t require rescue therapy from randomization through 7 days post procedure; this was met by 65% of patients the lusutrombopag group versus 29% of patients the control group.

The most common adverse event was headache, and the recommended dosage for lusutrombopag is 3 mg orally once daily with or without food for 7 days. Lusutrombopag is not indicated for general platelet normalization in patients with chronic liver disease and thrombocytopenia. Full prescribing information and further information about the approval can be found on the FDA website.

Lusutrombopag was approved in Japan in 2015 for this indication and is slated to be evaluated by the European Medicines Agency in 2019. The drug is marketed by Shionogi.

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