Press Release: Sanofi’s Wayrilz approved in the EU as the first BTK inhibitor to treat immune thrombocytopenia

Core Viewpoint - Sanofi's Wayrilz (rilzabrutinib) has been approved by the European Commission as the first BTK inhibitor for treating immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments, following a positive opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) [1][3] Group 1: Treatment Mechanism and Impact - Wayrilz addresses the underlying causes of ITP through multi-immune modulation, targeting various pathways in the immune system [2] - Traditional management of ITP focuses on restoring platelet counts, but Wayrilz offers a new approach by targeting the disease's root causes, potentially improving overall quality of life for patients [3][6] Group 2: Clinical Study and Results - The approval is based on the pivotal LUNA 3 phase 3 study, which demonstrated that Wayrilz met both primary and secondary endpoints, showing a positive impact on sustained platelet counts and other ITP symptoms [4][6] - In the LUNA 3 study, 64% of patients in the Wayrilz arm achieved platelet count response at 12 weeks, compared to 32% in the placebo arm [4] - Patients receiving Wayrilz reported a 10.6-point improvement in overall quality of life compared to a 2.3-point increase in the placebo group, based on the Immune Thrombocytopenia Patient Assessment Questionnaire [5] Group 3: Efficacy and Safety - Statistically significant durable platelet response was observed at week 25, with 23% of patients in the Wayrilz arm achieving this compared to 0% in the placebo arm (p<0.0001) [7] - The time to first platelet response was faster in the Wayrilz arm, averaging 36 days, while the placebo arm did not reach this endpoint [7] - Common adverse reactions (≥10% incidence) included diarrhea, nausea, headache, abdominal pain, and COVID-19 [5] Group 4: Regulatory Status and Future Prospects - Wayrilz has already been approved in the US and UAE, with ongoing regulatory reviews in Japan and China [8] - The drug has received fast track and orphan drug designations in the US for ITP, along with similar designations in the EU and Japan [8] - Sanofi is also investigating Wayrilz for additional rare diseases, including warm autoimmune hemolytic anemia, IgG4-related disease, and sickle cell disease [11]