The Food and Drug Administration (FDA) has approved Mulpleta (lusutrombopag; Shionogi) for the treatment of thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo a procedure.
The approval was based on data from 2 randomized, double-blind, placebo-controlled trials (L-PLUS 1 [N=97] and L-PLUS 2 [N=215]) involving patients with chronic liver disease who were undergoing an invasive procedure and had a platelet count <50 x 109/L. Patients were randomized to Mulpleta 3mg or placebo once daily for 7 days. In L‐PLUS 1, the major efficacy outcome was the proportion of patients who required no platelet transfusion prior to the primary invasive procedure, while in L‐PLUS 2, it was the proportion of patients who required no platelet transfusion prior to the primary invasive procedure and no rescue therapy for bleeding from randomization through 7 days after the primary invasive procedure. In both trials, responders were defined as patients who had a platelet count of ≥50 x 109/L with an increase of ≥20 x 109/L from baseline.
In L-PLUS 1, 78% of patients (38/49) receiving Mulpleta required no platelet transfusion prior to the invasive procedure, compared with 13% (6/48) who received placebo (treatment difference: 64%; P <.0001). Seventy-six percent of Mulpleta-treated patients were considered responders vs 6% of patients in the placebo arm (treatment difference: 68%; P <.0001).
In L-PLUS 2, 65% (70/108) of patients who received Mulpleta required no platelet transfusion prior to the invasive procedure or rescue therapy for bleeding through 7 days after the procedure, compared with 29% (31/107) receiving placebo (treatment difference: 37%; P <.0001). Sixty-five percent of Mulpleta-treated patients were considered responders vs 13% of patients in the placebo group (treatment difference: 52%; P <.0001).
The most common adverse reaction associated with therapy in clinical trials was headache.
Mulpleta, a thrombopoietin receptor agonist, will be supplied in 3mg strength tablets in blister packs containing 7 tablets.
For more information call (800) 849-9707 or visit Shionogi.com.