The U.S. Food and Drug Administration (FDA) has approved Wayrilz (rilzabrutinib) for adults with persistent or chronic immune thrombocytopenia (ITP) who did not respond adequately to prior treatment.
The approval is based on the pivotal LUNA 3 phase 3 trial, which demonstrated that Wayrilz significantly improved sustained platelet counts, reduced ITP symptoms, and enhanced health-related quality of life compared to placebo.
Wayrilz is a novel oral, reversible Bruton’s tyrosine kinase (BTK) inhibitor that works through multi-immune modulation, addressing key pathways driving ITP.
Durable platelet response at week 25: 23% with Wayrilz vs. 0% with placebo (p<0.0001)
Faster platelet response: median 36 days vs. not reached in placebo (p<0.0001)
Longer duration of response: 7 weeks vs. 0.7 weeks (placebo)
Improved quality of life: 10.6-point increase in Wayrilz arm vs. 2.3-point with placebo
The most common side effects (≥10%) included diarrhea, nausea, headache, abdominal pain, and COVID-19.
Experts note that Wayrilz provides a new treatment option for patients who fail steroids or do not respond to existing therapies, potentially changing the standard of care in ITP.
Approved in UAE (June 2025) for adults with persistent or chronic ITP.
Under review in the EU and China.
Holds FDA Fast Track and Orphan Drug Designations (ODD) for ITP, with additional ODD for wAIHA, IgG4-RD, and sickle cell disease.
Sanofi will support patients through its HemAssist program, which provides access guidance, financial assistance options, and educational resources for those living with rare blood disorders.