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] Group 1: Product Information - Wayrilz is the first BTK inhibitor for ITP, addressing the root cause of the disease through multi-immune modulation [5][7] - The drug has already received approval in the US and the UAE, and is under regulatory review in China [3] - Wayrilz has received fast track and orphan drug designations in the US, EU, and Japan for ITP and other rare diseases [3] Group 2: Clinical Study - 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] - The LUNA 3 study involved a randomized, multicenter design, comparing Wayrilz to placebo over a treatment period of 12 to 24 weeks [4] Group 3: Disease Context - Immune thrombocytopenia (ITP) is characterized by low platelet counts (<100,000/μL), leading to various bleeding symptoms and risks, including potentially life-threatening episodes [8] - Patients with ITP may experience reduced quality of life, including physical fatigue and cognitive impairment [8]

Sanofi’s Wayrilz recommended for EU approval by the CHMP to treat immune thrombocytopenia Recommendation based on LUNA 3 phase 3 study demonstrating rapid and durable platelet response and significant improvements in bleeding, quality of life measures, and other ITP symptoms   If approved, Wayrilz will be the first BTK inhibitor for ITP in the EU, targeting the root cause of the disease through multi-immune modulation Paris, October 17, 2025. The European Medicines Agency’s Committee for Medicinal Products ...

Core Insights - Sanofi has received FDA approval for Wayrilz (rilzabrutinib), marking it as the first BTK inhibitor approved for treating persistent or chronic immune thrombocytopenia (ITP) in the United States [1][7] - The approval was based on the phase III LUNA 3 study, which demonstrated positive outcomes in sustained platelet counts and ITP symptoms, achieving both primary and secondary endpoints [2][7] - Wayrilz is also under review in the EU and China for ITP treatment and has been recently approved in the UAE for the same indication [3] Company Developments - Patients using Wayrilz will benefit from Sanofi's HemAssist program, which offers support for treatments related to rare blood disorders [3] - In addition to ITP, Wayrilz is being developed for other rare diseases, including IgG4-related disease (IgG4-RD) and warm autoimmune hemolytic anemia (wAIHA), with orphan drug designations from the FDA [5][9] - A phase II study is currently evaluating Wayrilz for IgG4-RD, a chronic immune-mediated condition affecting multiple organs [8] Stock Performance - Sanofi's shares have increased by 2.6% this year, contrasting with a 0.6% decline in the industry [4]

Core Insights - The US FDA has approved Wayrilz (rilzabrutinib) for adults with persistent or chronic immune thrombocytopenia (ITP) who have not responded adequately to previous treatments, based on the successful LUNA 3 phase 3 study [1][3] - Wayrilz is a novel oral Bruton's tyrosine kinase (BTK) inhibitor that targets multiple immune pathways to address the underlying causes of ITP [2][11] - The approval highlights Sanofi's commitment to developing innovative therapies for rare and immunological diseases [3][11] Study Results - The LUNA 3 study involved 202 adult patients and demonstrated that 64% of patients on Wayrilz achieved a platelet count response at 12 weeks compared to 32% in the placebo group [3][10] - Patients on Wayrilz reported a 10.6-point improvement in health-related quality of life measures, while the placebo group showed a 2.3-point increase [4] - Statistically significant results included a durable platelet response at week 25 (23% in Wayrilz vs. 0% in placebo; p<0.0001) and a faster time to first platelet response (36 days in Wayrilz vs. not reached in placebo; p<0.0001) [7] Treatment Implications - Wayrilz offers a new treatment option for patients who have not responded to steroids or existing therapies, potentially improving management of ITP [5][11] - The drug has received Fast Track and Orphan Drug Designations from the FDA for ITP and is under regulatory review in the EU and China [8][12] - Sanofi's HemAssist program will provide support for patients undergoing treatment with Wayrilz, including assistance with access and insurance coverage [9] Company Overview - Sanofi is an R&D driven biopharma company focused on innovative therapies for various diseases, including rare and immunological conditions [13] - The company is committed to leveraging its understanding of the immune system to develop effective treatments and improve patient outcomes [13]

Core Insights - The FDA has approved Wayrilz (rilzabrutinib) as the first BTK inhibitor for adults with persistent or chronic immune thrombocytopenia (ITP) who have not responded adequately to previous treatments [1][11] - The approval is based on the LUNA 3 phase 3 study, which demonstrated significant improvements in platelet counts and other ITP symptoms [1][6] Company Overview - Sanofi is committed to addressing unmet patient needs through innovative therapies, with Wayrilz representing a significant advancement in the treatment of ITP [3][11] - The company utilizes its TAILORED COVALENCY® technology to selectively inhibit BTK, which plays a critical role in immune-mediated disease processes [11] Study Details - The LUNA 3 study involved 202 adult patients and showed that 64% of patients on Wayrilz achieved a platelet count response at 12 weeks compared to 32% in the placebo group [3][10] - Patients on Wayrilz reported a 10.6-point improvement in health-related quality of life measures, while the placebo group showed a 2.3-point increase [4] Treatment Mechanism - Wayrilz functions as a novel oral, reversible BTK inhibitor that targets multiple pathways in the immune system, addressing the root causes of ITP through multi-immune modulation [2][11] - This approach is expected to provide a new treatment option for patients who have not responded to traditional therapies [5][6] Regulatory Status - Wayrilz has received Fast Track and Orphan Drug Designations from the FDA for ITP, and similar designations in Japan and the EU [8] - The drug is also under regulatory review for ITP in the EU and China, indicating potential for broader market access [8] Patient Support - Sanofi offers the HemAssist patient support program to assist patients with treatment navigation, insurance coverage, and financial assistance [9]