The FDA has approved rilzabrutinib (Wayrilz) for the treatment of adult patients with persistent or chronic immune thrombocytopenia who have had an insufficient response to a previous treatment.1
The regulatory decision was supported by data from the pivotal phase 3 LUNA 3 trial (NCT04562766).2 Findings from LUNA 3 presented during the 2024 ASH Annual Meeting revealed that patients who received rilzabrutinib (n = 133) achieved a 25-week platelet response rate of 23% compared with 0% among those who received placebo (n = 69; P < .0001). Moreover, the time to first platelet response was 36 days in the investigational arm and was not reached in the placebo arm. The duration of platelet response was a least square mean of 7 weeks in the rilzabrutinib arm vs 0.7 weeks in the placebo arm.
“The burden of immune thrombocytopenia can be both physical and emotional with significant overlooked symptoms that can impact various aspects of daily living,” Caroline Kruse, president and chief executive officer at the Platelet Disorder Support Association, stated in a news release.1 “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.”
In terms of safety, the most common adverse effects that occurred in at least 10% of patients included diarrhea, nausea, headache, abdominal pain, and COVID-19. Patients in the rilzabrutinib arm experienced a 10.6-point improvement across 9 health-related quality of life measures per The Immune Thrombocytopenia Patient Assessment Questionnaire; patients in the control arm had a 2.3-point increase.
References
- Sanofi’s Wayrilz approved in US as first BTK inhibitor for immune thrombocytopenia. News release. Sanofi. August 29, 2025. Accessed September 2, 2025. https://www.news.sanofi.us/2025-08-29-Sanofis-Wayrilz-approved-in-US-as-first-BTK-inhibitor-for-immune-thrombocytopenia
- Kuter DJ, Ghanima W, Cooper N, et al. Safety and efficacy of rilzabrutinib vs placebo in adults with immune thrombocytopenia: the phase 3 LUNA3 study. Blood. 2025;145(24):2914-2926. doi:10.1182/blood.2024027336