Wayrilz (rilzabrutinib) / Sanofi - LARVOL DELTA

Treatment outcomes with rilzabrutinib in participants with allergic comorbidities: a post hoc analysis of a phase 2a study in IgG4-related disease (ACR Convergence 2026) - No abstract available

Clinical • P2a data • Retrospective data • Inflammation

Baseline disease characteristics in IgG4-related disease study participants with and without flare: post hoc analysis of a phase 2a study of rilzabrutinib (ACR Convergence 2026) - No abstract available

P2a data • Retrospective data • Inflammation

Beyond Omalizumab: Emerging Biologic and Small Molecule Therapies in Chronic Spontaneous Urticaria. (PubMed, Clin Exp Allergy) - "The oral BTK inhibitor remibrutinib is now licensed for adults in the United States and European Union; Dupilumab, approved from 12 years of age in 2025, was extended to children aged 2-11 years in 2026 on basis of extrapolated efficacy supported by paediatric pharmacokinetic and safety data. Barzolvolimab has completed Phase 3 enrolment of 1939 adults, briquilimab, EVO756, EP262 and rilzabrutinib are advancing in adults and ligelizumab failed to demonstrate superiority over omalizumab...Adults dominate the trial populations; adolescents are enrolled as underpowered subgroups; dedicated paediatric evidence is essentially confined to dupilumab; and older adults-in whom comorbidity, polypharmacy and the cardiovascular and malignancy signals of JAK inhibition matter most-are almost never analyzed separately. Head-to-head and sequencing trials, endotype-stratified patient selection, age-inclusive trial design and long-term safety data are the immediate priorities if..."

Journal • Review • Cardiovascular • Chronic Spontaneous Urticaria • Dermatology • Immunology • Oncology • Pediatrics • Urticaria • IL13 • IL4

A subgroup analysis of rilzabrutinib efficacy on asthma control in a phase 2 asthma study (ERS 2026) - P2 | "Rilzabrutinib demonstrated clinically meaningful improvement in asthma control across subgroups based on baseline demographics, disease characteristics, and biomarkers."

Clinical • P2 data • Asthma • Immunology • Respiratory Diseases

Clinical activity and safety of rilzabrutinib in patients with previously treated immune thrombocytopenia (LUNA3): results from the open-label period of a phase 3 trial. (PubMed, Lancet Haematol) - P3 | "Rilzabrutinib showed continued, stable, and sometimes improved platelet responses, including in patients who were initially non-responsive in the double-blind trial. Additionally, rilzabrutinib improved several disease aspects, including physical fatigue and bleeding, supporting its multi-immune modulation mechanism in immune thrombocytopenia, and had a favourable safety profile. The long-term extension period of LUNA3 will further characterise the long-term efficacy and safety of rilzabrutinib in patients with difficult-to-treat immune thrombocytopenia, and the ongoing LUNA4 trial (NCT07007962) will determine the efficacy of rilzabrutinib in earlier-line treatment and its potential to achieve sustained treatment-free response."

Journal • P3 data • Cardiovascular • Cytomegalovirus Infection • Fatigue • Hematological Disorders • Hypertension • Immune Modulation • Immune Thrombocytopenic Purpura • Immunology • Interstitial Lung Disease • Pulmonary Disease • Respiratory Diseases • Thrombocytopenia • Thrombocytopenic Purpura

Emerging pharmacotherapies for chronic spontaneous urticaria: spotlight on BTK inhibitors. (PubMed, Expert Opin Pharmacother) - "BTK inhibitors act on intracellular pathways involved in mast-cell activation, basophil signaling, and B-cell mediated immune responses. Studies have shown reductions in disease activity across multiple BTK inhibitors, with benefit observed early after treatment initiation in several studies. Remibrutinib is the first BTK inhibitor approved for CSU and has the most extensive phase 3 evidence. Oral administration may provide a treatment option for patients. Defining the place of BTK inhibitors within treatment algorithms and identifying patients most likely to benefit from this approach remain areas for future research."

Journal • Review • Chronic Spontaneous Urticaria • Dermatology • Immunology • Urticaria

A phase 2 trial of rilzabrutinib in IgG4-related disease. (PubMed, Ann Rheum Dis) - P2 | "Rilzabrutinib had an acceptable safety profile in patients with IgG4-related disease who had failed/ intolerant to rituximab and who were rituximab-naïve. Most participants discontinued GCs successfully without disease flares and exhibited sustained disease activity reduction. Further investigation of this potential alternative to B-cell-depleting therapies is needed in larger, long-term, placebo-controlled trials."

Journal • P2 data • Infectious Disease • Inflammation • Novel Coronavirus Disease • Respiratory Diseases

Wayrilz: Data readout from P3 RILIEF trial (NCT07190196) for IgG4-related disease in 2028 (Sanofi) - Q2 2026 Results: Data readout from P3 LUMINA-3 trial (NCT07086976) for warm autoimmune hemolytic anemia in 2028

P3 data • Autoimmune Hemolytic Anemia • Immunology

Wayrilz: Regulatory submissions in US/EU for IgG4-related disease in 2028 (Sanofi) - Q2 2026 Results: Regulatory submissions in US/EU for warm autoimmune hemolytic anemia in 2028

EMA filing • FDA filing • Autoimmune Hemolytic Anemia • Immunology

Network meta-analysis comparing the efficacy of subsequent treatment regimens in primary immune thrombocytopenia patients (ISTH 2026) - "In placebo- controlled studies, Romiplostim showed the highest efficacy, followed by Hetrombopag, Eltrombopag, Fostamatinib, and Rilzabrutinib. In studies using Danazol as the control, ATRA plus danazol had the highest efficacy, followed by Tacrolimus plus danazol and rhTPO plus danazol. In studies using Eltrombopag as the control, the Eltrombopag plus diacerein regimen was more effective than rhTPO. Conclusions These results furnish high-quality evidence to support evidence-based, individualized selection of subsequent therapy for adults with relapsed/refractory primary immune thrombocytopenia. Table or Figure Upload (1) Figure 1.Network meta-analysis comparing the efficacy of subsequent treatment regimens Page 2 DOI*10.1016/j.rpth.2026.104395"

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

Immune thrombocytopenia: Evolving mechanistic understanding and clinical development of new therapies. (PubMed, Tzu Chi Med J) - "Although corticosteroids, intravenous immunoglobulin, anti-D immunoglobulin, rituximab, splenectomy, and thrombopoietin receptor agonists have constituted the therapeutic foundation for decades, these approaches remain limited by transient responses, toxicity, and insufficient disease modification...Bruton tyrosine kinase (BTK) inhibitors (rilzabrutinib and orelabrutinib) modulate B-cell activation and macrophage effector pathways; spleen tyrosine kinase inhibitors (fostamatinib) block Fcγ receptor-mediated phagocytosis, neonatal Fc receptor inhibitors (efgartigimod and rozanolixizumab) accelerate IgG catabolism and rapidly reduce pathogenic autoantibody burden; and complement inhibition (sutimlimab) provides a rational strategy for patients in whom classical pathway activation contributes to refractory disease. Collectively, these agents represent the most significant expansion of the ITP treatment landscape in decades, offering steroid-sparing options and enabling more..."

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

Bruton Tyrosine Kinase as Therapeutic Target in Cardiovascular Diseases-Executive Summary. (PubMed, Thromb Haemost) - "Indeed, highly selective Btk inhibitors (BTKi) show no or only minor bleeding in autoimmune disease trials, and rilzabrutinib, approved in 2025, reduces bleeding in patients with immune thrombocytopenia (ITP). This executive summary of a recent state-of-the-art review synthesizes current understanding of Btk's mechanistic contributions to thrombosis and CVD, evaluates the evolution of BTKi, and explores their therapeutic potential."

Journal • Cardiovascular • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Oncology • Primary Immunodeficiency • Thrombocytopenia • Thrombocytopenic Purpura • Thrombosis • BTK

Impact of Bruton Tyrosine Kinase Inhibitor (BTKi) Rilzabrutinib on Cardiovascular Events in Adults With Persistent/Chronic Primary Immune Thrombocytopenia (ITP): An Integrated Analysis of The Phase 2 and Phase 3 LUNA Studies (ISTH 2026) - P2, P3 | "Conclusions Rilzabrutinib demonstrated low cardiovascular AE rates with no increase vs placebo in LUNA3 DB, and maintained very low rates with prolonged exposure, supporting a favorable cardiovascular safety profile in adults with persistent/chronic ITP. Page 2 Table or Figure Upload (1) Table or Figure Upload (2) Page 3 DOI*10.1016/j.rpth.2026.103586"

Clinical • P2 data • P3 data • Cardiovascular • Diabetes • Dyslipidemia • Hematological Disorders • Hypertension • Immune Thrombocytopenic Purpura • Metabolic Disorders • Thrombocytopenia • Thrombocytopenic Purpura

Impact of BTK Inhibitor Rilzabrutinib on Menstrual Health Outcomes in Women With Persistent/Chronic Primary Immune Thrombocytopenia (ITP) During the Open-Label and Long-Term Extension Periods of the Phase 3 LUNA3 Multicenter Study (ISTH 2026) - P3 | "Results support the potential for additional, clinically- meaningful benefits from long-term, continued treatment with rilzabrutinib, a BTK inhibitor acting through multi-immune modulation. Page 2 Table or Figure Upload (1) DOI*10.1016/j.rpth.2026.104386"

Clinical • HEOR • P3 data • Hematological Disorders • Immune Modulation • Immune Thrombocytopenic Purpura • Immunology • Thrombocytopenia • Thrombocytopenic Purpura • Women's Health

LUMINA 3: A Study to Investigate the Efficacy, Safety, and Pharmacokinetics of Oral Rilzabrutinib Compared With Placebo in Participants 18 Years of Age and Older With Warm Autoimmune Hemolytic Anemia (clinicaltrials.gov) - P3 | N=90 | Sponsor: Sanofi

Trial initiation date • Anemia • Autoimmune Hemolytic Anemia • Hematological Disorders • Immunology

The Medicines and Healthcare products Regulatory Agency (MHRA) has today (29 May 2026) granted a marketing authorisation for the medicines rilzabrutinib (Wayrilz) to treat adults with immune thrombocytopenia (ITP) when prior treatments for ITP have not worked well enough. (GOV.UK) - "This approval is based on a pivotal phase 3 study of 202 patients with ITP whose prior treatments had been insufficient."

MHRA approval • Immunology • Thrombocytopenia

The Ministry of Health, Labour and Welfare in Japan has granted marketing and manufacturing authorization to Wayrilz (rilzabrutinib), a novel oral reversible Bruton’s tyrosine kinase inhibitor (BTKi), for the treatment of persistent or chronic immune thrombocytopenia (GlobeNewswire) - "The approval is based on the LUNA 3 phase 3 study (clinical study identifier: NCT04562766), in which Wayrilz met the primary and secondary endpoints, showing a positive impact on sustained platelet counts and other symptoms."

Japan approval • Immune Thrombocytopenic Purpura • Immunology • Thrombocytopenia

Time to Meaningful Clinical Response Across Approved and Emerging Therapies for Antihistamine-Refractory Chronic Spontaneous Urticaria: A Network Meta-Analysis. (PubMed, J Clin Med) - " All drugs except rilzabrutinib 400 mg daily demonstrated faster mean time to MCID than placebo. Fenebrutinib had the fastest mean time to MCID (0.67-0.76 weeks), and tezepelumab the slowest (5.41-5.65 weeks)...Omalizumab, dupilumab, and ligelizumab demonstrated statistically significant reductions in time to MCID compared with placebo. Head-to-head trials with standardized outcome reporting would enable more definitive comparative conclusions."

Journal • Retrospective data • Chronic Spontaneous Urticaria • Dermatology • Immunology • Urticaria

CHARACTERIZING EARLY AND LATE RESPONDERS TO MULTI-IMMUNE MODULATOR RILZABRUTINIB IN PERSISTENT/CHRONIC IMMUNE THROMBOCYTOPENIA: FINDINGS FROM THE PHASE 3 LUNA3 STUDY (EHA 2026) - P3 | "No clear clinical factors, including the number of prior ITP treatments or other baseline features, distinguished early from late responders. Further investigation is needed to identify potential predictors of early and late response."

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

PHASE 3 LUNA3 FINAL EFFICACY AND SAFETY RESULTS FOR RILZABRUTINIB IN THE LONG-TERM EXTENSION PERIOD IN ADULTS WITH PERSISTENT/CHRONIC IMMUNE THROMBOCYTOPENIA (ITP) (EHA 2026) - P3 | "A high percentage of pts reduced or discontinued concomitant ITP therapy. Rilzabrutinib continues to be well tolerated, with a favorable safety profile in adults with persistent/chronic ITP."

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

Wayrilz (rilzabrutinib, Sanofi) Positioned to Define Third-Line Treatment in Warm Autoimmune Hemolytic Anemia (w-AIHA) as IMAAVY (niopcalimab, Jonhson & Johnson) Follows Closely with Positive Data Announcement at EHA 2026 (GlobeNewswire) - "Findings from the newly released Market Dynamix: AIHA (US), 2026 study from Spherix Global Insights reveal growing physician anticipation for late-stage pipeline agents....The study, which surveyed 71 U.S. hematologists managing patients with AIHA, found that corticosteroids remain the overwhelming first-line standard, while rituximab is firmly established in second line....Among the late-stage pipeline agents evaluated, rilzabrutinib (currently marketed as Wayrilz for immune thrombocytopenia [ITP], Sanofi), nipocalimab (currently marketed as Imaavy for generalized myasthenia gravis [gMG], Johnson & Johnson) and ianalumab (Novartis) are contributing to growing physician optimism that the w-AIHA treatment landscape may soon evolve beyond longstanding reliance on steroids and rituximab."

Clinical data • Autoimmune Hemolytic Anemia

Justifying score interpretation thresholds for the Asthma Control Questionnaire (ACQ-5) in a contemporary asthma patient population. (PubMed, J Patient Rep Outcomes) - P2 | "Contemporary score interpretation methods support a MWPC threshold of ≥ 0.6, which is functionally identical to the extant threshold of ≥ 0.5, given the 0.2 increments of the ACQ-5 scale. MBGD ranged from 0.2 to 0.6, further supporting use of the established ≥ 0.5 threshold to define clinically important change between groups of patients. A "well-controlled" threshold of ≤ 0.75 was also supported."

Journal • Asthma • Immunology • Pulmonary Disease • Respiratory Diseases

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) - P2 | "In a phase 2a trial, 70% of participants treated with rilzabrutinib monotherapy and a rapid prednisone taper were flare free without additional treatments (glucocorticoids or immunosuppressants)...Methods The phase 2a (NCT04520451) multicenter, open-label, 52-week clinical trial of oral rilzabrutinib 400 mg given twice daily, enrolled participants refractory or intolerant to rituximab and participants naïve to rituximab...Specifically, BTK inhibition may have effects on the underlying processes driving excess IgG4 production that is characteristic of the disease, without major impact on IgG- dependent aspects of the immune response. An ongoing multinational phase 3 study shall further investigate these preliminary findings in a larger patient population."

P2a data • Inflammation

Sustained response after rilzabrutinib discontinuation in multirefractory immune thrombocytopenia. (PubMed, Blood Vessel Thromb Hemost) - No abstract available

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

INT22854: A study to investigate a potential effect of rilzabrutinib (SAR444671) on ethinylestradiol and levonorgestrel from combined oral contraceptives in healthy female participants (clinicaltrialsregister.eu) - P1 | N=24 | Not yet recruiting | Sponsor: Sanofi-Aventis Recherche & Developpement

New P1 trial • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura