FDA Approval Announcement
On July 31, 2018, the U.S. Food and Drug Administration approved lusutrombopag (Mulpleta, Shionogi Inc.) for the treatment of thrombocytopenia in adults with chronic liver disease who are scheduled to undergo a medical or dental procedure.
Clinical Trial Basis for Approval
The approval was based on two randomized, double‑blind, placebo‑controlled trials (L‑PLUS 1 and L‑PLUS 2, NCT02389621) enrolling a total of 312 patients with chronic liver disease and severe thrombocytopenia (platelet count < 50 × 10⁹/L) who were scheduled for an invasive procedure.Patients were randomized 1:1 to receive either lusutrombopag 3 mg or placebo once daily for up to 7 days.
Efficacy Results
In L‑PLUS 1: 78% (38/49) of patients in the lusutrombopag group required no platelet transfusion before the primary invasive procedure, compared with 13% (6/48) in the placebo group (95% CI: 49%, 79%; p<0.0001).
In L‑PLUS 2: 65% (70/108) of patients receiving lusutrombopag required no platelet transfusion before the procedure or rescue therapy for bleeding through 7 days post‑procedure, versus 29% (31/107) in the placebo group (95% CI: 25%, 49%; p<0.0001).
Adverse Reactions
The most common adverse reaction reported in ≥ 3% of patients was headache.
Recommended Dosage
The recommended dosage of lusutrombopag is 3 mg orally once daily, with or without food, for 7 days.
Regulatory Designations
The FDA granted this application priority review and fast track designation. Information on FDA expedited programs is available in the Guidance for Industry: Expedited Programs for Serious Conditions—Drugs and Biologics.
