Dive Brief:
- The Food and Drug Administration this week approved Shionogi's Mulpeta for thrombocytopenia in patients with chronic liver disease (CLD) who scheduled to undergo a medical or dental procedure.
- The drug is a small molecule medication designed to reduce the risk for bleeding events after a surgical procedure in CLD patients with thrombocytopenia.
- Approval was based on positive results from two double-blind, placebo-controlled clinical trials with 312 patients, including the multinational global Phase 3 study known as L-PLUS2. Previously, platelet transfusions were the only treatment option for patients with CLD and thrombocytopenia undergoing non-emergency invasive procedures.
Dive Insight:
Approval of Mulpeta (lusutrombopag) fills a critical treatment need for patients with CLD and associated thrombocytopenia. In fact, CLD-associated thrombocytopenia is one of the most common hematological complications of CLD, and one of the most expensive.
Those with CLD-associated thrombocytopenia, or very low platelet count, are at increased risk not only for bleeding events after medical procedures, but also may require recurrent platelet transfusion. The annual healthcare costs for patients with hepatitis C and CLD who also have thrombocytopenia is three times that of patients with hepatitis C and CLD without thrombocytopenia.
In the two clinical trials that led to Mulpeta’s approval, patients were randomly assigned to receive placebo or 3 mg of once daily for up to 7 days prior to undergoing a medical procedure.
Results from the L-PLUS1 trial showed 78% of patients treated with Mulpeta required no platelet transfusion before the medical procedure, compared with 13% who received placebo. And in L-PLUS2, 65% of patients who received the study medication did not require a platelet transfusion or rescue therapy for bleeding from the time of randomization until 7 days after the procedure. However, 29% of those who received placebo needed a platelet transfusion or rescue therapy.
Across the trials, Mulpeta was well tolerated, and the most common adverse event was headache. The overall results of L-PLUS2 revealed that 5.6 % of patients taking Mulpeta experienced adverse events, while 12.1% of those on placebo had side effects or complications. What's more, adverse events related to bleeding occurred in 2.8% of patients on Mulpeta vs 5.6% of patients in the L-PLUS2's placebo group. Adverse events involving thrombosis occurred in 1.9% of patients in both the Mulpeta and placebo groups.