Press Release: Sanofi's Wayrilz recommended for EU approval by the CHMP to treat immune thrombocytopenia

Core Viewpoint - The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of Wayrilz (rilzabrutinib) for treating immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments, with a final decision expected soon [1][2] Company Overview - Sanofi is an R&D driven biopharma company focused on improving lives through innovative medicines and vaccines, leveraging a deep understanding of the immune system [9] Product Information - Wayrilz is the first BTK inhibitor for ITP, addressing the root cause of the disease through multi-immune modulation, and is already approved in the US and the UAE [5][3] - The drug has received fast track and orphan drug designations in the US, EU, and Japan for ITP and is under regulatory review in China [3] Clinical Study Insights - The positive CHMP opinion is based on the 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 [2][4] - LUNA 3 involved a randomized, multicenter design, with patients receiving either Wayrilz or placebo over a treatment period of 12 to 24 weeks [4] Disease Context - ITP is characterized by low platelet counts (<100,000/μL), leading to various bleeding symptoms and a risk of thromboembolism, significantly affecting patients' quality of life [8]