Sanofi Wayrilz wins USFDA approval for adults with immune thrombocytopenia
Paris: Sanofi has received approval from the US Food and Drug Administration (FDA) for Wayrilz (rilzabrutinib) for adults with persistent or chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.
The approval was based on the pivotal LUNA 3 phase 3 study, in which Wayrilz met the primary and secondary endpoints, showing a positive impact on sustained platelet counts and other ITP symptoms.
“The burden of immune thrombocytopenia can be both physical and emotional with significant overlooked symptoms that can impact various aspects of daily living,” said Caroline Kruse, President and CEO at the Platelet Disorder Support Association. “We are pleased to have a new treatment option that can help ease the ongoing strain of managing the disease for patients and their families."
As a novel oral, reversible, Bruton’s tyrosine kinase (BTK) inhibitor, Wayrilz can help address the root causes of ITP through multi-immune modulation, targeting different pathways across the immune system.
“With its differentiated mechanism of action, Wayrilz has the potential to become a treatment of choice for immune thrombocytopenia patients who have not responded to a prior therapy,” said Brian Foard, Executive Vice President, Head of Specialty Care at Sanofi. “Its multi-immune modulation approach shows promise in addressing the key drivers of immune thrombocytopenia, which aligns with Sanofi's commitment to adapting and evolving therapeutic solutions to help tackle ongoing unmet patient needs. This approval underscores Sanofi's expertise and ambitions at the junction of rare and immunological disease."
The LUNA 3 phase 3 study, presented at the 66th American Society of Hematology Annual Meeting and Exposition, evaluated the efficacy and safety of Wayrilz compared to placebo in adults (n=202) with persistent or chronic ITP. Patients who achieved platelet count response at 12 weeks were eligible to continue the full 24-week double-blind period (64% of patients in the Wayrilz arm and 32% of patients in the placebo arm). Patients receiving Wayrilz experienced the following compared to patients receiving placebo:
- Statistically significant durable platelet response at week 25 (23% of patients in Wayrilz arm vs. 0% in placebo arm; p<0.0001)
- Faster time to first platelet response (36 days in Wayrilz arm vs. not reached in placebo arm; p<0.0001)
- Longer duration of platelet response (least square mean of 7 weeks in Wayrilz arm vs. 0.7 weeks in placebo arm)
Patients receiving Wayrilz reported an overall 10.6-point improvement across nine health-related quality of life measures compared to a 2.3-point increase in the placebo arm, based on The Immune Thrombocytopenia Patient Assessment Questionnaire, a clinical tool designed to measure ITP symptoms. The results of this analysis are descriptive and were not powered for statistical significance.
“Traditionally, immune thrombocytopenia management has focused on restoring platelet counts and reducing bleeding risk, which for some patients may result in suboptimal responses, persistent symptoms, or unacceptable treatment complications,” said David Kuter, MD, Director of Clinical Hematology at Massachusetts General Hospital and Professor of Medicine at Harvard Medical School, study author. “Through multi-immune modulation, Wayrilz can offer a new option for patients, including those who fail steroids or do not respond to existing treatment.”
Wayrilz was approved in the United Arab Emirates for adult patients with persistent or chronic ITP who have had an insufficient response or intolerance to a previous treatment in June 2025. Wayrilz is currently under regulatory review for ITP in the EU and China.
It received Fast Track and Orphan Drug Designations (ODD) from the FDA for ITP, with similar orphan designations in Japan and the EU. Most recently, the FDA granted Wayrilz ODD for three additional rare diseases, including warm autoimmune hemolytic anemia (wAIHA), IgG4-related disease (IgG4-RD), and sickle cell disease (SCD). Wayrilz also received FDA Fast Track Designation and European Medicines Agency orphan designation in IgG4-RD.
