Chronic Liver Disease Treatment: Mulpleta Now Available Across the United States
Chronic liver disease patients scheduled for medical procedures now have access to a new FDA approved treatment option. Shionogi announced the U.S. availability of Mulpleta (lusutrombopag), a once daily oral medication designed to address thrombocytopenia in adult patients with chronic liver disease who are preparing to undergo a procedure. This approval represents a meaningful advance for a patient population that has historically had limited options beyond platelet transfusions.
At FOMAT Medical Research, hepatology and chronic liver disease are among the therapeutic areas where we actively conduct clinical trials. FDA approvals like this one are the direct result of the Phase 3 clinical trial process that organizations like FOMAT support every day.
What Is Chronic Liver Disease and Why Does Thrombocytopenia Matter?
Chronic liver disease encompasses a broad range of conditions that progressively damage liver function over time, including cirrhosis, hepatitis, and nonalcoholic fatty liver disease. One of the most common and serious complications of chronic liver disease is thrombocytopenia, a condition in which platelet counts drop to dangerously low levels.
Low platelet counts are particularly problematic when patients need to undergo surgical or interventional procedures. Platelets are essential for blood clotting, and patients with chronic liver disease who have thrombocytopenia face a significantly elevated risk of uncontrolled bleeding during and after procedures. Until recently, the primary management strategy was platelet transfusion, which carries its own risks and logistical challenges.
Mulpleta offers physicians and patients an alternative that can be administered orally at home in the days leading up to a procedure, potentially eliminating the need for transfusions altogether.
What Is Mulpleta and How Does It Work?
Mulpleta (lusutrombopag) is a small molecule thrombopoietin (TPO) receptor agonist. TPO is a hormone that stimulates the bone marrow to produce platelets. By activating the TPO receptor, Mulpleta signals the body to increase platelet production, helping patients with chronic liver disease reach platelet levels sufficient to safely undergo procedures.
The medication is taken once daily and is orally administered, making it significantly more convenient than transfusion based approaches. It is designed specifically for adult patients with chronic liver disease who are scheduled to undergo a procedure and have thrombocytopenia.
“As adult patients with chronic liver disease often undergo procedures that could put them at increased risk for bleeding, this treatment will offer physicians and patients an option other than platelet transfusions,” said Takayuki Yoshioka, president and CEO of Shionogi Inc.
FDA Approval Based on Phase 3 Clinical Trial Data
The U.S. FDA approved Mulpleta on July 31, 2018. The approval was based on consistent safety and efficacy data from two Phase 3 clinical trials, L-PLUS 1 and L-PLUS 2, in which Mulpleta met both primary and secondary endpoints with statistically significant results across both studies.
This kind of rigorous Phase 3 evidence is the foundation of every FDA approval. The L-PLUS trials demonstrated that Mulpleta could reliably raise platelet counts in chronic liver disease patients to levels that reduced or eliminated the need for platelet transfusions before procedures.
Mulpleta Assist: Patient Support Program
Alongside the product launch, Shionogi introduced Mulpleta Assist, a full service patient access and support program. Mulpleta Assist provides healthcare providers and patients with dedicated resources to manage medication logistics, coordinate managed care reimbursement, match patients with financial assistance programs, and offer overall patient support throughout the treatment process.
The goal of Mulpleta Assist is to ensure that patients who need Mulpleta can access it and receive the full benefit of the therapy, regardless of insurance or financial barriers.
What This Means for Clinical Research in Hepatology
The approval of Mulpleta highlights the ongoing need for clinical trial infrastructure that can support complex hepatology studies involving patients with chronic liver disease. These patients often present with comorbidities and require careful procedural management, making experienced site networks essential for successful trial execution.
FOMAT Medical Research has extensive experience supporting clinical trials in digestive diseases and hepatology, including studies involving patients from diverse and underserved communities where chronic liver conditions are highly prevalent. Our national network of community based investigators is equipped to enroll and manage patients with chronic liver disease across Phase 1 through Phase 4 studies. Contact our team to learn how FOMAT can support your next hepatology trial, or explore our digestive diseases capabilities.