Rystiggo (rozanolixizumab-noli) / UCB - LARVOL DELTA

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

Neonatal fc receptor antagonists in adult patients with primary immune thrombocytopenia: Systematic review and meta-analysis of randomized controlled trials (ASH 2025) - "Several FcRn inhibitors(namely efgartigimod, rozanolixizumab, and batoclimab) have shown promising results in clinical trials,but to date, no meta-analysis has synthesized randomized trial data to evaluate their efficacy and safetyin ITP.MethodsWe conducted a systematic review and meta-analysis of randomized, placebo-controlled trials evaluatingFcRn antagonists in adult patients with ITP. Despite the small number of short-duration trials and modest sample sizes, theconsistency of treatment effect and favorable safety profile support FcRn blockade as a promising andgenerally well-tolerated treatment option. Larger, longer-term studies are needed to confirm thedurability of response and better characterize the long-term safety profile."

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

Depletion and recovery of IgG following treatment with Rozanoliximab and Imlifidase in pigtail macaques. (PubMed, bioRxiv) - "Here, we report in simian immunodeficiency virus (SIV) infected pigtail macaques ( Macaca nemestrina ) the capacity of two orthogonal strategies to systemically deplete IgG - treatment with a neonatal Fc receptor blocking antibody (Rozanolixizumab) that restricts IgG rescue and recycling, and administration of the IgG protease Imlifidase (IdeS) that cleaves the Fc domain...We observed a similar degree of depletion, comparable kinetics of rebound among SIV antigen-specific fractions as total IgG, but differential balance among IgG subclasses following treatment in some cases. In sum, this study in a nonhuman primate model describes the efficacy and downstream impacts of new tools to modify humoral immune states providing insight into the balance between protective and pathological effects of IgG antibodies."

Journal • ACACA

Phase 4 Study Evaluating Efficacy and Safety of Rozanolixizumab in Adult Chinese Participants With Generalized Myasthenia Gravis (clinicaltrials.gov) - P4 | N=40 | Not yet recruiting | Sponsor: UCB Biopharma SRL

New P4 trial • CNS Disorders • Myasthenia Gravis

Pharmacovigilance analysis of FcRn antagonists in the treatment of myasthenia gravis: A disproportionality analysis based on the FAERS database. (PubMed, Hum Vaccin Immunother) - "This study aimed to evaluate the safety profiles of FcRn antagonists, efgartigimod alfa and rozanolixizumab, in the treatment of myasthenia gravis using real-world adverse event data from the FAERS database. The study identifies novel safety signals and highlights clinically relevant risk patterns, supporting enhanced monitoring in clinical use. It is crucial to emphasize that this disproportionality analysis is exploratory in nature and identifies potential associations, which do not establish causality and require confirmation through further dedicated studies."

Adverse events • Journal • Atrial Fibrillation • Cardiovascular • CNS Disorders • Genito-urinary Cancer • Infectious Disease • Myasthenia Gravis • Nephrology • Oncology • Pain • Prostate Cancer • Sleep Disorder • Solid Tumor

Complement C5 Inhibitor (Anti-C5) and Neonatal Fc Receptor Antagonist Anti-FcRn in Myasthenia Gravis at University Public Hospitals of Paris, France (AP-HP): What Is the Market Share of These New Treatments? (ISPOR-EU 2025) - "This falls to 352 DDD in 2023 with the arrival of ravulizumab, then goes to 1861 DDD in 2024 despite the eculizumab biosimilar. Prescriptions are tending towards efgartigimod-IV, though ravulizumab continues to hold an important place, particularly in long-stay patients, where it replaces eculizumab and generates the highest costs. However, this trend could change with the arrival of subcutaneous forms available in community pharmacies (zilucoplan, efgartigimod-SC and rozanolixizumab), potentially modifying therapeutic practices and hospital costs."

Clinical • CNS Disorders • Myasthenia Gravis

Cost-Effectiveness of Rozanolixizumab vs. Efgartigimod in the Treatment of Generalized Myasthenia Gravis (gMG) in Greece (ISPOR-EU 2025) - "Rozanolixizumab + SoC offers more QALYs at an incremental saving over a lifetime horizon for the third-party payer in Greece compared to efgartigimod + SoC in gMG patients who remain symptomatic on SoC alone."

Cost effectiveness • HEOR • CNS Disorders • Myasthenia Gravis

Budget Impact Analysis of Rozanolixizumab for the Treatment of Generalized Myasthenia Gravis (gMG) in Greece (ISPOR-EU 2025) - "OBJECTIVES: To assess the budget impact of reimbursing rozanolixizumab as a treatment for adult patients with moderate-to-severe generalized myasthenia gravis (gMG) in Greece from the perspective of the third-party payer, namely the National Organization for Health Services Provision (EOPYY). A budget impact model (BIM) was developed over a five-year time horizon (2025-2029) to estimate the cost of treating eligible gMG patients with existing therapies, including eculizumab, efgartigimod, ravulizumab, rituximab, intravenous immunoglobulin, or rozanolixizumab. The reimbursement of rozanolixizumab for gMG in Greece is projected to result in measurable savings on the third-party payer's budget for gMG over five years. This positive budgetary impact, combined with rozanolixizumab's mechanism of action, mode of administration and clinical benefits confirms rozanolixizumab as a valuable gMG treatment option in Greece."

HEOR • CNS Disorders • Myasthenia Gravis

Rozanolixizumab as rescue therapy in triple-seronegative refractory generalized myasthenia gravis. (PubMed, Immunol Med) - "Fast-acting therapies, such as intravenous methylprednisolone (IVMP), intravenous immunoglobulin (IVIG) and plasma exchange (PLEX), are typically recommended during disease exacerbation. This case, with long-term follow-up, suggests the potential utility of rozanolixizumab as rescue therapy in a patient subgroup which is typically excluded from randomized controlled trials. Larger studies specifically including patients with triple-seronegative MG are warranted."

Journal • CNS Disorders • Dyslipidemia • Immunology • Myasthenia Gravis

Measuring the effect of rozanolixizumab using the Myasthenia Gravis Impairment Index: analyses from the randomized phase 3 MycarinG study. (PubMed, J Neurol) - P3 | "These findings further support efficacy analyses from MycarinG to highlight the benefit of rozanolixizumab in patients with gMG and demonstrate the utility of the MGII in evaluating patient-relevant symptoms following treatment."

Clinical • Journal • P3 data • CNS Disorders • Immunology • Myasthenia Gravis

Management of Myasthenic Crisis and Emerging Roles of Molecularly Targeted Therapies: A Narrative Review. (PubMed, Neurol Int) - "Conventional MCr treatments-such as plasma exchange (PLEX), intravenous immunoglobulin (IVIG), and intravenous methylprednisolone (IVMP)-remain standard treatments; however, they present significant limitations, including delayed onset of action, adverse effects, and inconsistent efficacy...We review published case reports involving FcRn inhibitors (efgartigimod, efgartigimod-SC, rozanolixizumab) and complement inhibitors (eculizumab, ravulizumab, zilucoplan), highlighting their rapid onset of action and safety profiles in MCr...We also discuss strategic considerations for therapy selection, including antibody subtype, coexisting autoimmune conditions, genetic factors, and transition to long-term maintenance. Though the current evidence is promising, large-scale randomized studies are needed to establish definitive roles for these therapies in MCr management."

Journal • Review • CNS Disorders • Critical care • Immunology • Myasthenia Gravis • Respiratory Diseases

A total of 18 abstracts will be presented at AANEM and the MGFA scientific session, highlighting UCB's dedication to driving advancements in gMG treatment. (PRNewswire) - "These presentations will cover data on rozanolixizumab and zilucoplan, including new insights on corticosteroid-sparing strategies and quality of life, and long-term safety and effectiveness data. Additionally, insights from a real-world study on user characteristics and adherence patterns from the HumaMG app will be shared, reinforcing UCB's commitment to improving gMG management."

Clinical data • Myasthenia Gravis

Safety profile of complement C5 inhibitors and FcRn inhibitors in the treatment of myasthenia gravis: analysis of the FAERS database and disease-gene interaction network. (PubMed, Front Immunol) - "To integrate pharmacovigilance and network pharmacology methods for a comprehensive analysis of the potential adverse reactions of complement C5 inhibitors (eculizumab, ravulizumab, zilucoplan) and neonatal Fc receptor (FcRn) inhibitors (efgartigimod, rozanolixizumab), and to explore their toxicity mechanisms, thereby providing a reference for rapidly understanding the safety of these two novel classes of biologics in the treatment of myasthenia gravis (MG). Furthermore, network pharmacology analysis revealed potential mechanisms underlying these adverse reactions. These findings provide valuable insights for monitoring and managing risks during treatment with two novel classes of biologics."

Journal • Cardiovascular • CNS Disorders • Congestive Heart Failure • Dyspepsia • Gastric Cancer • Genito-urinary Cancer • Heart Failure • Hepatology • Immunology • Inflammatory Arthritis • Ischemic stroke • Liver Failure • Myasthenia Gravis • Nephrology • Oncology • Peripheral Arterial Disease • Prostate Cancer • Psoriatic Arthritis • Renal Calculi • Rheumatology • Seronegative Spondyloarthropathies • Solid Tumor

From promise to practice: evaluating the clinical impact of FcRn inhibition in IgG-mediated autoimmune rheumatic diseases. (PubMed, Front Immunol) - "Several FcRn-targeting agents, such as efgartigimod, rozanolixizumab, and nipocalimab, have demonstrated favorable safety and efficacy profiles in clinical trials and are now approved or under investigation for multiple indications. This review also explored personalized therapeutic approaches, combination strategies, and the future landscape of FcRn-targeted drug development. While FcRn inhibition offered a paradigm shift in managing antibody-driven diseases, long-term safety and patient stratification remain key challenges for future research."

Journal • Review • Immunology • Inflammatory Arthritis • Lupus • Rheumatoid Arthritis • Rheumatology • Systemic Lupus Erythematosus • Vasculitis

Blocking the neonatal Fc receptor as a novel approach to prevent cardiac neonatal lupus: a proof-of-concept study. (PubMed, Ann Rheum Dis) - "The successful application of FcRn blockade to prevent recurrent cardiac-NL sets a precedent for a multicentre study."

Journal • Immunology • Inflammatory Arthritis • Lupus • Systemic Lupus Erythematosus

EFFICACY AND SAFETY OF ROZANOLIXIZUMAB TREATMENT CYCLES IN PATIENTS WITH GENERALISED MYASTHENIA GRAVIS: FINAL POOLED ANALYSIS OF PHASE 3 STUDIES (WCN 2025) - P3 | "Rozanolixizumab showed consistent improvements across multiple MG-specific outcomes up to 13 cycles and was generally well tolerated following repeated cyclic treatment."

P3 data • Retrospective data • CNS Disorders • Myasthenia Gravis

TREATMENT GUIDELINES AND COMPLEMENT INHIBITORS (WCN 2025) - "Components available in Japan includes eculizumab, ravulizumab and zilucoplan as C5 inhibitors, efgartigimod and rozanolixizumab as FcRn inhibitors. This prevents the cleavage of C5 into C5a and C5b and thus prevents formation of the membrane attack complex. Zilucoplan also binds to the domain of C5 that corresponds to C5b to block interaction with complement component C6 (dual action)."

CNS Disorders • Myasthenia Gravis

THE ROLE OF FCRN ANTAGONISTS IN TRANSFORMING THE MYASTHENIA GRAVIS TREATMENT PARADIGM (WCN 2025) - "Early clinical trials of FcRn antagonists, such as efgartigimod, rozanolixizumab, and nipocalimab, have shown significant potential, including the reduction of symptom severity, rapid clinical improvement, and enhanced quality of life for patients with generalized myasthenia gravis (gMG). Furthermore, a Phase III clinical trial conducted in China for another FcRn antagonist, batoclimab, has also demonstrated positive results, further confirming the effectiveness and safety of this class of medications. The advent of FcRn antagonists marks a significant milestone in the management of MG, opening a new chapter of precision therapy in autoimmune neurology and offering a promising treatment paradigm for other IgG-mediated diseases."

CNS Disorders • Myasthenia Gravis

Self-administration of rozanolixizumab via manual push and infusion pump methods in patients with generalised myasthenia gravis: a randomised, phase 3, open-label, crossover study. (PubMed, J Neurol) - P3 | "All patients successfully self-administered rozanolixizumab; more patients preferred manual push. Efficacy and safety were consistent with the known HCP-administered profile. These results support rozanolixizumab self-administration and manual push administration in patients with gMG."

Clinical • Journal • P3 data • CNS Disorders • Myasthenia Gravis

IPND 2025: Revised Consensus Criteria, Classification, and Nomenclature for Neuromyelitis Optica Spectrum Disorders (ECTRIMS 2025) - "S Mariotto served on advisory board for UCB, received received support from TSF and GJF, is site PI for clinical trials of Rozanolixizumab (UCB) and Satralizumab (Roche) in MOGAD, and received speaker honoraria from UCB, Novartis, Biogen, Sanofi, Alexion, Roche, TSF, Amgen, Dynamics...S Pittock has received personal compensation for consulting or serving on scientific advisory or data safety monitoring boards for Arialys, Astellas, F. Hoffmann-La Roche AG, Genentech, Sage Therapeutics, and UCB; his institution has received compensation for his serving as a consultant for Alexion, Amgen, Arialys, and Astellas; research support from Adimmune, Alexion, Amgen, F. Hoffmann-La Roche AG, and Genentech; and has 2 patents issued (8889102; application 12-678350; Neuromyelitis Optica Autoantibodies as a Marker for Neoplasia; and 9891219B2; application 12-573942; Methods for Treating Neuromyelitis Optica [NMO] by Administration of Eculizumab to an Individual that is Aquaporin-4..."

IO biomarker • Late-breaking abstract • CNS Disorders • Multiple Sclerosis • Neuromyelitis Optica Spectrum Disorder • Novel Coronavirus Disease • Rare Diseases • Solid Tumor

Models to predict prognosis in an international multicenter study of myelin oligodendrocyte glycoprotein antibody-associated disease (ECTRIMS 2025) - "[Study Funded by UCB] Disclousure of interest: Dr Flanagan has served on an advisory board for UCB, has received research support from UCB, is site principal investigator for clinical trials of Rozanolixizumab and Satralizumab in MOGAD, funded by UCB and Roche, respectively...M Isabel Leite has received support for scientific and educational meetings from UCB.Dr Sean Pittock has received personal compensation for consulting or serving on scientific advisory or data safety monitoring boards for Arialys, Astellas, F. Hoffmann-La Roche AG, Genentech, Sage Therapeutics, and UCB; his institution has received compensation for his serving as a consultant for Alexion, Amgen, Arialys, and Astellas; research support from Adimmune, Alexion, Amgen, F. Hoffmann-La Roche AG, and Genentech; and has 2 patents issued (8889102; application 12-678350; Neuromyelitis Optica Autoantibodies as a Marker for Neoplasia; and 9891219B2; application 12-573942; Methods for Treating Neuromyelitis..."

Clinical • Late-breaking abstract • CNS Disorders • Multiple Sclerosis • Neuromyelitis Optica Spectrum Disorder • Ophthalmology • Pediatrics • Rare Diseases • Solid Tumor

Future treatments for myelin oligodendrocyte glycoprotein antibody-associated disease: the clinical trial landscape. (PubMed, Expert Opin Emerg Drugs) - "This review explores the evolving treatment landscape of adult MOGAD, with a focus on immunotherapies under active clinical investigation: azathioprine, tocilizumab, satralizumab, and rozanolixizumab. In the next five years, one or more of these agents may achieve regulatory approval, particularly if biomarker-driven strategies and trial designs are refined. Addressing unmet needs in pediatric populations and low-resource settings will be essential to ensure equitable, personalized treatment."

IO biomarker • Journal • Review • CNS Disorders • Immunology • Multiple Sclerosis • Neuromyelitis Optica Spectrum Disorder • Pediatrics • Rare Diseases • Solid Tumor

Aseptic Meningitis Following Rozanolixizumab in a Patient With Refractory Anti-MuSK Positive Myasthenia Gravis: A Case Report. (PubMed, Neurohospitalist) - "A 36-year-old woman with refractory anti-MuSK-positive generalized myasthenia gravis received an infusion of rozanolixizumab after failing to respond to corticosteroids and azathioprine. Clinicians should be aware of this potential adverse effect and consider drug-induced etiologies in similar clinical scenarios. Early recognition may prevent unnecessary treatment and hospitalizations."

Journal • CNS Disorders • Infectious Disease • Myasthenia Gravis • Pain

Enhanced Access to gMG Treatments: UCB Canada Secures pCPA Agreements for ZILBRYSQ and RYSTIGGO (Canada Newswire) - "This significant milestone comes after successfully securing a positive reimbursement recommendation by CDA-AMC and recognition of therapeutic value by INESSS and is a necessary step for securing provincial public reimbursement as the next and final step."

Reimbursement • Myasthenia Gravis

Improvement in Patient-Reported Symptoms of Generalised Myasthenia Gravis With Rozanolixizumab in the Randomised Phase 3 MycarinG Study Using the MG Symptoms PRO. (PubMed, Eur J Neurol) - P3 | "MG Symptoms PRO scales correlate well with concepts in MG-ADL while assessing additional concepts, such as PF and MWF. Results from the MG Symptoms PRO in MycarinG reflected improvements from baseline in patient-relevant symptoms, including fatigue, with rozanolixizumab."

Clinical • Journal • P3 data • CNS Disorders • Fatigue • Myasthenia Gravis