Wayrilz (rilzabrutinib) / Sanofi - LARVOL DELTA

Serum IgG4:IgG ratio in participants with and without flare: an exploratory analysis of a phase 2a study of rilzabrutinib in participants with IgG4-related disease (EULAR 2026) - No abstract available

P2a data • Inflammation

MODIFIED DURABLE PLATELET RESPONSE BY INTERNATIONAL WORKING GROUP CRITERIA: LUNA3 PHASE 3 STUDY OF RILZABRUTINIB VS PLACEBO IN ADULTS WITH PRIMARY IMMUNE THROMBOCYTOPENIA (THSNA 2026) - P3 | "Modified durable IWG response in patients randomized to rilzabrutinib during the double-blind period was achieved in 19 patients receiving 1-2 prior unique ITP therapies, which included 17 (89%) patients receiving prior corticosteroids, 7 (37%) TPO-RA, 2 (11%) IVIg, and 1 (5%) each splenectomy, immunosuppressants and other immunomodulatory agents (including cyclophosphamide), and/or other. Overall, modified durable IWG response was increased in rilzabrutinib-treated patients during the double-blind period; some non-durable responders to either double-blind rilzabrutinib or placebo had improved response during the open-label period. Results also suggest higher responses in patients with fewer prior ITP therapies. No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author."

Clinical • P3 data • Hematological Disorders • Immune Modulation • Immune Thrombocytopenic Purpura • Immunology • Thrombocytopenia • Thrombocytopenic Purpura

REDUCTION IN CONCOMITANT THERAPY USE WITH RILZABRUTINIB AND SUSTAINED RESPONSE IN ADULTS WITH PERSISTENT/CHRONIC IMMUNE THROMBOCYTOPENIA (ITP) IN THE PHASE 3 LUNA3 LONG-TERM EXTENSION (LTE) PERIOD (THSNA 2026) - P3 | "Ten LTE patients discontinued corticosteroids (switched to rilzabrutinib monotherapy), 2 reduced corticosteroids ≥50% from DB baseline, and 6 reduced corticosteroid dose to <5 mg (prednisone qd)...Of 11 patients on concomitant TPO-RA or corticosteroid/TPO-RA at DB baseline, 5 discontinued TPO-RA before entering LTE (romiplostim: n=1; avatrombopag: n=2; eltrombopag: n=2) and 1 during the LTE (eltrombopag)... Rilzabrutinib continued to demonstrate favorable safety, sustained platelet responses and improved ITP-related symptoms during the LTE period. Most LTE patients receiving concomitant corticosteroids and/or TPO-RA were able to discontinue/reduce use while maintaining platelet counts. These results further underscore the disease-modifying potential of multi-immune modulation with rilzabrutinib in patients with ITP."

Clinical • P3 data • Hematological Disorders • Immune Modulation • Immune Thrombocytopenic Purpura • Immunology • Infectious Disease • Thrombocytopenia • Thrombocytopenic Purpura

104: Immune Thrombocytopenia: Management Strategies for the Medical Enigma (HOPA 2026) - "Recently the novel BTK inhibitor, rilzabrutinib, joined the armamentarium for ITP-directed therapy. In this session we will evaluate the FDA-approved ITP treatments. UAN: 0465-0000-26-017-L01-P Knowledge or Application Based: Knowledge Learning Objectives: Outline the pathogenesis and clinical presentation of immunethrombocytopenia (ITP)​Recognize the manifestations of ITP and complications of itstreatment​Evaluate safety and efficacy outcomes for the management of ITP ​Design a treatment plan for a patient with chronic ITP"

Immune Thrombocytopenic Purpura • Immunology • Thrombocytopenia • Thrombocytopenic Purpura

Autoimmune Bullous Diseases: Therapeutic Update. (PubMed, Drugs) - "More specifically, RCTs with immunoadsorption, rilzabrutinib, and efgartigimod in pemphigus, as well as dupilumab, benralizumab, mepolizumab, avdoralimab, and efgartigimod in bullous pemphigoid are summarized. In addition, an overview is given about the current treatment landscape on the basis of national and international guidelines as well the experience of the authors. In all, while licensing of rituximab for pemphigus vulgaris and dupilumab for bullous pemphigoid represents a therapeutic breakthrough, the lack of industry-sponsored RCTs in mucous membrane pemphigoid and the failure of recent RCTs in pemphigus and bullous pemphigoid demonstrate, on the one hand, the high medical need for effective and safe therapies in AIBDs and, on the other hand, the apparent difficulty in finding suitable compounds and appropriate trial designs."

Journal • Review • Bullous Pemphigoid • Dermatology • Dermatopathology • Immunology • Pemphigus Vulgaris • IL13

Efficacy of Rilzabrutinib on Asthma Quality of Life: Pooled Results From a Double-blind Placebo-controlled Phase 2 Study (ATS 2026) - No abstract available

Clinical • HEOR • P2 data • Asthma • Immunology • Respiratory Diseases

LPS18573: A study to test how well and how safe an oral medication called rilzabrutinib is for adults with a condition called immune thrombocytopenia (ITP) who did not respond to their initial treatment including corticosteroids, intravenous immunoglobulin, and/or anti-D immunoglobulin (LUNA 4) (clinicaltrialsregister.eu) - P2/3 | N=43 | Recruiting | Sponsor: Sanofi-Aventis Recherche & Developpement

New P2/3 trial • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

Press Release: Sanofi’s rilzabrutinib earns orphan drug designation in Japan for IgG4-related disease (GlobeNewswire) - "Rilzabrutinib for the treatment of IgG4-RD was evaluated in a phase 2 study (clinical study identifier: NCT04520451) and results were presented at the European Alliance of Associations for Rheumatology 2025 congress."

Orphan drug • Immunology • Inflammation

Efficacy of Rilzabrutinib on Asthma Control Based on Prior Exacerbation History: Pooled Results From a Double-Blind, Placebo-Controlled, Phase 2 Study (ATS 2026) - No abstract available

Clinical • P2 data • Asthma • Immunology • Respiratory Diseases

A Study of Rilzabrutinib in Adult Patients With Immune Thrombocytopenia (ITP) (clinicaltrials.gov) - P2 | N=86 | Completed | Sponsor: Principia Biopharma, a Sanofi Company | Active, not recruiting ➔ Completed

Trial completion • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

Translational immunothrombosis in autoimmune Heparin-Induced thrombocytopenia: targeting the FcγRIIa-Syk-BTK and complement pathways. (PubMed, Clin Exp Med) - "Preclinical and clinical data indicate that targeting these axes with Syk inhibitors (fostamatinib), BTK inhibitors (rilzabrutinib, zanubrutinib), and complement inhibitors (sutimlimab) can restore platelet counts and mitigate immune-driven thrombosis. These findings underscore the therapeutic potential of pathway-specific interventions in aHIT, highlighting the need for biomarker-guided, translational trials to validate their efficacy and safety. Bridging mechanistic evidence from ITP and APS provides a framework for precision immunotherapy in autoimmune HIT."

IO biomarker • Journal • Review • Cardiovascular • Genetic Disorders • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Thrombocytopenia • Thrombocytopenic Purpura • Thrombosis • SYK

Rilzabrutinib: “The Committee was reminded of the status of this application and its remaining outstanding issues”; Chronic immune thrombocytopenia (European Medicines Agency) - CHMP Final Minutes of the meeting on 21 - 24 Jul 2025: “The Committee adopted a list of outstanding issues with a specific timetable”

CHMP • Immunology • Thrombocytopenia

Press Release: Sanofi’s rilzabrutinib designated breakthrough therapy in the US and orphan drug in Japan for the treatment of warm autoimmune hemolytic anemia (The Manila Times) - "Both designations are based on clinical data from the ongoing LUMINA 2 phase 2b study (clinical study identifier: NCT05002777) assessing the efficacy and safety of rilzabrutinib for patients with wAIHA. In addition, the new LUMINA 3 phase 3 study (clinical study identifier: NCT07086976), is assessing rilzabrutinib compared with placebo in patients with wAIHA."

Breakthrough therapy • Orphan drug • Autoimmune Hemolytic Anemia

2025: strong sales and EPS growth. (GlobeNewswire) - "ALTUVIIIO (haemophilia A) sales were €324 million of which 85% was in the US...Rest of World sales of €48 million benefited from the launches in Japan and Taiwan...Rezurock (chronic graft-versus-host disease) sales were €113 million and decreased by 6.1%...Sales in Europe were -€4 million, mainly from a one-time credit in the UK. In Rest of World, sales were €12 million, the overwhelming majority from the launch in China; Cablivi (acquired thrombotic thrombocytopenic purpura) sales were €69 million and increased by 1.4%, driven by more patients being treated in the US and Europe offset by an element of price impact in the US....Wayrilz (immune thrombocytopenia) sales were €6 million, all in the US, following approval in August 2025; Qfitlia (haemophilia A and B) sales were €4 million, all in the US, following approval in March 2025."

Sales • Chronic Graft versus Host Disease • Hemophilia A • Hemophilia B • Immune Thrombocytopenic Purpura

Properties of FDA-approved small molecule protein kinase inhibitors: a 2026 update. (PubMed, Pharmacol Res) - "The following ten drugs received FDA approval in 2025 - avutometinib (inhibiting MEK1/2 in serous ovarian carcinomas), defactinib (blocking FAK in low grade serous ovarian carcinomas), delgocitinib (antagonizing the JAK family in hand eczema), mirdametinib (inhibiting MEK1/2 in type I neurofibromatosis), remibrutinib (blocking BTK in chronic spontaneous urticaria), rilzabrutinib (antagonizing BTK in chronic immune thrombocytopenia), sunvozertinib (blocking mutant exon 21 insertion EGFR NSCLC), taletrectinib (inhibiting mutant ROS1 in NSCLC), vimseltinib (blocking CSF1R in tenosynovial giant cell tumors), and zongertinib (antagonizing mutant HER2 in NSCLC). This article summarizes the physicochemical properties of all 94 FDA-approved small molecule protein kinase inhibitors including the molecular weight, number of hydrogen bond donors/acceptors, ligand efficiency, lipophilic efficiency, polar surface area, and solubility. A total of 45 of the 94 FDA-approved drugs have a..."

FDA event • Journal • Review • Atopic Dermatitis • Chronic Spontaneous Urticaria • Contact Dermatitis • Dermatology • Genetic Disorders • Giant Cell Tumor of Bone • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Lung Cancer • Neurofibromatosis • Non Small Cell Lung Cancer • Oncology • Ovarian Cancer • Ovarian Serous Adenocarcinoma • Solid Tumor • Tenosynovial Giant Cell Tumor • Thrombocytopenia • Thrombocytopenic Purpura • Urticaria • CSF1R • EGFR • HER-2 • ROS1

Wayrilz: Regulatory decision in Japan for immune thrombocytopenic purpura in H2 2026 (Sanofi) - Q4 & FY2025 Results

Japan approval • Immune Thrombocytopenic Purpura • Immunology

Immune thrombocytopenia: contemporary pathophysiology, treatment gaps, and the role of novel mechanisms in patient-centered care. (PubMed, Am J Manag Care) - P3 | "Rilzabrutinib targets several aspects of ITP disease pathophysiology by modulating multiple immune pathways. Approval was based on results from a phase 3 trial (LUNA 3 [NCT04562766]), in which patients with ITP who received rilzabrutinib demonstrated a rapid, durable platelet response and improvements in fatigue and bleeding with a tolerable safety profile."

Journal • Review • Fatigue • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Thrombocytopenia • Thrombocytopenic Purpura

Rilzabrutinib in immune thrombocytopenia: a targeted, patient-centered alternative to conventional therapies. (PubMed, Ann Med Surg (Lond)) - No abstract available

Journal • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

Press Release: Sanofi’s Wayrilz approved in the EU as the first BTK inhibitor to treat immune thrombocytopenia (The Manila Times) - "The EU approval of Wayrilz is based on the pivotal LUNA 3 phase 3 study (clinical study identifier: NCT04562766), in which Wayrilz met the primary and secondary endpoints, demonstrating a positive impact on sustained platelet counts as well as other ITP symptoms....Wayrilz has already been approved in the US and the United Arab Emirates (UAE), and it is currently under regulatory review for ITP in Japan and China."

China filing • EMA approval • Japan filing • Immune Thrombocytopenic Purpura

Rilzabrutinib for Chronic Immune Thrombocytopenia (ASH 2025) - No abstract available

Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

Clinical Discussant for Chronic Immune Thrombocytopenia (ASH 2025) - "Discuss the clinical relevance of and possible applications for novel therapy in ITP (rilzabrutinib)"

Clinical • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

Feasibility assessment of indirect treatment comparison between off-label rituximab and novel treatments in patients with warm autoimmune hemolytic anemia (ASH 2025) - "Among the clinical trials included, five studied rituximab, in combination with prednisone, prednisolone, ibrutinib, or bortezomib. Three trials studied fostamatinib, while other studied treatments included pegcetacoplan, sovleplenib, parsaclisib, and rilzabrutinib...Future work should consider de novo sources of real-world evidence for rituximab that more closely align with registrational trial characteristics and endpoint definitions. However, aligning timing of endpoint measurements between registration trials and real-world data to match definitions remains challenging."

Clinical • Anemia • Autoimmune Hemolytic Anemia • Hematological Disorders • Immunology

Comparative effectiveness of second-line therapies for chronic immune thrombocytopenia: A network meta-analysis of RCTs (ASH 2025) - "The treatment groups were: Avatrombopag (96), Eltrombopag (283), Fostamatinib (101), Rilzabrutinib (133), Romiplostim (2116), and Rozanolixizumab (41)...Rituximab was analyzed indirectly and showed moderate efficacy... This NMA provides a comparative analysis of second-line therapies for chronic ITP, with Rozanolixizumab identified as the most effective treatment. Despite variability in safety profiles, all therapies demonstrated significant clinical benefits, supporting the adoption of personalized treatment strategies."

HEOR • Retrospective data • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura • SYK

Fatigue, hemoglobin, and inflammatory markers in warm autoimmune hemolytic anemia: Analysis from a phase 2b trial of rilzabrutinib (LUMINA2) (ASH 2025) - "Correlation of Hct changes with inflammatory markers and fatigue were measured and the relationship assessed. BTK inhibition shows a major effect on Hct and inflammatory markers in the treatment of wAIHA and shows great promise in addressing this disorder"

P2b data • Anemia • Autoimmune Hemolytic Anemia • Fatigue • Hematological Disorders • Immunology

Long-term efficacy and safety of rilzabrutinib, an oral bruton tyrosine kinase (BTK) inhibitor in patients with warm autoimmune hemolytic anemia (wAIHA) in the LUMINA Phase 2b part B study: A 74-week follow-up (ASH 2025) - P2 | "Extended follow-up of pts treated with rilzabrutinib showed continued efficacy with sustainedHb response, with some pts achieving drug-free remission; decreased hemolytic markers; clinicallymeaningful improvement in fatigue score; and a favorable safety profile in pts with wAIHA. Efficacy andsafety of rilzabrutinib will be further evaluated in a phase 3, randomized, placebo-controlled study(LUMINA3)."

Clinical • P2b data • Anemia • Autoimmune Hemolytic Anemia • Back Pain • Cardiovascular • Hematological Disorders • Immune Modulation • Immunology • Infectious Disease • Inflammation • Musculoskeletal Pain • Respiratory Diseases • Thrombosis