The US Food and Drug Administration (FDA) has approved 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-III 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. 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," said Caroline Kruse, President and CEO, Platelet Disorder Support Association.

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. Its multi-immune modulation approach shows promise in addressing the key drivers of immune thrombocytopenia, which aligns with Sanofi's commitment to adapt and evolve 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," said Brian Foard, Executive Vice President, Head – Specialty Care, Sanofi.  

The LUNA 3 phase-III 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 percent of patients in the Wayrilz arm and 32 per cent of patients in the placebo arm).

Patients treated with Wayrilz demonstrated better outcomes compared to those receiving placebo, according to data released alongside the US FDA approval. At week 25, 23 percent of patients in the Wayrilz arm achieved a statistically significant durable platelet response versus none in the placebo group (p<0.0001). The median time to first platelet response was also faster at 36 days, compared with ‘not reached’ in the placebo arm (p<0.0001). The duration of platelet response averaged seven weeks with Wayrilz versus less than one week for placebo.

In addition to efficacy, patients reported meaningful improvements in quality of life. Based on The Immune Thrombocytopenia Patient Assessment Questionnaire, those receiving Wayrilz saw a 10.6-point improvement across nine health-related quality of life measures, compared with a 2.3-point increase in the placebo arm. These results were descriptive and not powered for statistical significance.

The most common adverse reactions observed in ≥10 per cent of patients were diarrhoea, nausea, headache, abdominal pain and COVID-19.

“Traditionally, immune thrombocytopenia management has focused on restoring platelet counts and reducing bleeding risk, which, for some patients, may result in sub-optimal responses, persistent symptoms, or unacceptable treatment complications.  Through multi-immune modulation, Wayrilz can offer a new option for patients, including those who fail steroids or do not respond to the existing treatment,” said David Kuter, MD, Director–Clinical Hematology, Massachusetts General Hospital and Professor–Medicine, Harvard Medical School, and study author.

Wayrilz had previously been approved in the United Arab Emirates (UAE) in June 2025 for adults with persistent or chronic ITP who had an insufficient response or intolerance to prior therapy. Regulatory reviews are ongoing in the European Union (EU) and China.

The therapy has also received multiple designations from the FDA, including Fast Track and Orphan Drug Designation (ODD) for ITP. Additional ODDs were recently granted for three other rare diseases—warm autoimmune hemolytic anemia (wAIHA), IgG4-related disease (IgG4-RD), and Sickle Cell Disease (SCD). In IgG4-RD, Wayrilz has also been granted FDA Fast Track Designation and EMA orphan designation.

Patients prescribed Wayrilz in the US will be able to access Sanofi’s HemAssist programme, which provides support across the company’s rare blood disorder portfolio. The programme helps patients navigate insurance coverage, financial assistance and educational resources.