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

Core Viewpoint - The European Commission has approved Wayrilz (rilzabrutinib), 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: Product Overview - Wayrilz is a novel, oral, reversible BTK inhibitor that targets the underlying causes of ITP through multi-immune modulation, addressing various pathways in the immune system [2][10] - The approval is based on the pivotal LUNA 3 phase 3 study, which demonstrated a positive impact on sustained platelet counts and other ITP symptoms [3][6] Group 2: Clinical Study Insights - The LUNA 3 study involved 202 adult patients with persistent or chronic ITP, showing that 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 most common adverse reactions (incidence ≥10%) included diarrhea, nausea, headache, abdominal pain, and COVID-19 [5] Group 4: Regulatory Status and Future Prospects - Wayrilz has also been approved in the US and UAE, with ongoing regulatory reviews in Japan and China, and has received fast track and orphan drug designations in the US and EU for ITP [8][10] - The drug is being studied for additional rare diseases, including warm autoimmune hemolytic anemia, IgG4-related disease, and sickle cell disease [11]