The second member of the FDA-approved thrombopoietin receptor agonist drug class was proven efficacious and safe in a pair of phase 3 trials.
Dr. Tsutae Nagata
The US Food and Drug Administration (FDA) has approved lusutrombopag (Mulpleta) for the treatment of thrombocytopenia in particular adults with chronic liver disease (CLD).
The thrombopoietin receptor agonist (TPO RA) therapy, from Shionogi Inc., was approved on Monday for the treatment of low blood-platelet count in adult patients with chronic liver disease who are also scheduled to undergo a medical or dental procedure.
The approval—the second of the TPO RA drug class following the FDA’s nod to Dova Pharmaceutical’s own form of the therapy in May—was based on the results of 2 randomized, double-blind, placebo-controlled trials (L-PLUS 1 and 2) which involved 300-plus patients with chronic liver disease and severe thrombocytopenia.
In L-PLUS 1, 97 patients with CLD—as defined by platelet counts <50,000 mcL—who were to undergo invasive procedure were randomized to receive 3 mg lusutrombopag or placebo. Researchers reported that 78% (n= 38) of treated patients did not require platelet transfusion prior to procedure, while just 13% (6) of patients treated with placebo did not require a transfusion.
In L-PLUS 2, 215 patients with CLD were again randomized to receive 3 mg lusutrombopag or placebo for up to 7 days, with invasive procedures performed between Day 9 and 14. Researchers found 64.8% of treated patients required platelet transfusion prior to the procedure, versus just 29% of placebo patients. Adverse events related to bleeding were prevalent in 2.8% of treated patients and 5.6% of placebo patients.
Each group reported a thrombosis-related adverse event rate of 1.9%.
At the time of the announced trial results in September 2017, Dr. Tsutae Nagata, chief medical officer of Shionogi, noted that patients with CLD and thrombocytopenia undergoing non-emergent invasive procedures had no approved medical treatment options aside from platelet transfusions.
“Therefore therapeutic options in this area are critically needed,” Nagata said in a statement.
Shionogi did not release any statement at the time of the FDA approval.