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]