Sanofi has received the US Food and Drug Administration (FDA) approval for Wayrilz (rilzabrutinib) to treat adults with chronic immune thrombocytopenia (ITP), for whom previous treatments are inadequate.
Wayrilz is an oral Bruton’s tyrosine kinase (BTK) inhibitor that works by modulating immune responses to address the underlying causes of ITP.
The FDA approval follows Fast Track and Orphan Drug Designations for ITP and three other rare diseases, including warm autoimmune haemolytic anaemia, IgG4-related disease, and sickle cell disease.
In addition to the US, Wayrilz has been approved in the United Arab Emirates (UAE) for similar indications and is undergoing regulatory reviews in the European Union (EU) and China.
Sanofi executive vice president and speciality care head Brian Foard said: “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 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 FDA approval is based on the Phase 3 LUNA 3 study, which showed that Wayrilz met its primary and secondary endpoints, improving sustained platelet counts and other ITP symptoms.
The LUNA 3 study, involving 202 adult patients, highlighted Wayrilz’s effectiveness compared to placebo, with significant improvements in platelet response time and duration.
Patients treated with Wayrilz reported improved quality of life measures. The most common adverse effects include diarrhoea, nausea, and headaches.
Furthermore, Sanofi is offering the HemAssist programme to support patients and caregivers with access to Wayrilz, insurance navigation, and financial assistance.
LUNA 3 study author David Kuter said: “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.
“Through multi-immune modulation, Wayrilz can offer a new option for patients, including those who fail steroids or do not respond to existing treatment.”
In July 2025, Sanofi agreed to acquire Vicebio, a UK-based biotechnology company, for up to $1.6bn.
