Rystiggo (rozanolixizumab-noli) / UCB - LARVOL DELTA

Rozanolixizumab: A Review in AChR or MuSK Autoantibody-Positive Generalised Myasthenia Gravis. (PubMed, CNS Drugs) - "Rozanolixizumab was generally well tolerated with an acceptable safety profile; the most common adverse events were headache, diarrhoea, pyrexia and nausea. Thus, rozanolixizumab is an efficacious and generally well tolerated therapy for adults with AChR or MuSK Ab+ gMG, representing a convenient and valuable additional treatment option for these patients."

Journal • Review • CNS Disorders • Immunology • Myasthenia Gravis • Pain

Comparative efficacy, safety, and functional recovery of neonatal fc receptor inhibitors and conventional immunotherapies for chronic inflammatory demyelinating polyradiculoneuropathy: a systematic review and network meta-analysis. (PubMed, Front Immunol) - "Interventions included FcRn inhibitors (efgartigimod alfa, rozanolixizumab), intravenous/subcutaneous immunoglobulin (IVIg/SCIG), corticosteroids, plasma exchange (PLEX), and rituximab. IVIg remains a broadly supported comparator across induction and maintenance settings. These findings should inform, but not replace, individualized clinical decision-making because patient-level treatment-effect modification, biomarker status, prior treatment response, and long-term neurophysiological outcomes were not directly evaluated."

Clinical • IO biomarker • Journal • Retrospective data • Review • Pain

Comparative Efficacy of Rozanolixizumab with Efgartigimod or Intravenous Immunoglobulin in Generalized Myasthenia Gravis Using Matching Adjusted Indirect Comparisons. (PubMed, Neurol Ther) - "Without direct comparative evidence, these analyses provide additional context to inform treatment decisions for the management of gMG. These analyses suggest that FcRn inhibitors represent important targeted treatment options for gMG."

Journal • CNS Disorders • Immunology • Myasthenia Gravis

A Study to Evaluate The Long-Term Safety And Efficacy of Rozanolixizumab in Adult Participants With Ocular Myasthenia Gravis (clinicaltrials.gov) - P3 | N=100 | Recruiting | Sponsor: UCB Biopharma SRL | Not yet recruiting ➔ Recruiting

Enrollment open • CNS Disorders • Immunology • Myasthenia Gravis

Regimen-stratified Bayesian model-based meta-analysis of targeted biologics in generalised myasthenia gravis: efficacy comparison, mechanism-level class contrast, and endpoint sensitivity with model-informed trial design. (PubMed, Naunyn Schmiedebergs Arch Pharmacol) - "The study was registered with PROSPERO: CRD420261288549. On MG-ADL, zilucoplan led with the highest Emax (4.8 points; 95% CrI: 4.2-5.4) and largest net benefit (2.5; 2.0-3.1)...Efgartigimod achieved the fastest onset on both scales (≤ 1.6 weeks)...For rozanolixizumab on MG-ADL, snapshot week 26 endpoints were underpowered (65%); integrated endpoints recovered power to 94-95%...Across the multi dimensional treatment-selection framework integrating efficacy, safety, tolerability, and convenience, batoclimab emerged as the highest-ranked agent in all eight clinical priority profiles. These findings provide a pharmacodynamic evidence base for mechanism-guided treatment individualisation and endpoint optimisation in generalised myasthenia gravis."

Journal • Retrospective data • CNS Disorders • Immunology • Myasthenia Gravis

Efficacy and safety of rozanolixizumab in severe fibromyalgia: a phase 2A randomised, double-blind, placebo-controlled, multicentre trial. (PubMed, Lancet Rheumatol) - P2 | "Rozanolixizumab did not demonstrate broad efficacy in this severe fibromyalgia population. While there was an improvement in BPI-SF, this was not meaningful at a group level. Inability to select for patients with pathogenic autoantibodies might have contributed to between-patient heterogeneity. Response-predictors are required to determine whether IgG reduction benefits subsets of patients with severe fibromyalgia."

Journal • P2a data • CNS Disorders • Cognitive Disorders • Fatigue • Fibromyalgia • Musculoskeletal Pain • Pain • Rheumatology • Sleep Disorder

Practical Guidance on Initiating and Switching Targeted Immunotherapies in Generalised Myasthenia Gravis: A German-Austrian Expert Opinion Paper. (PubMed, Eur J Neurol) - "This expert statement provides a practice-oriented framework integrating current evidence and clinical experience to support individualised therapeutic decision-making in gMG."

Journal • CNS Disorders • Immunology • Myasthenia Gravis • Pediatrics

Clinical Course After a Single Cycle of Rozanolixizumab in Generalized Late-Onset Myasthenia Gravis. (PubMed, J Clin Neurol) - No abstract available

Journal • CNS Disorders • Immunology • Myasthenia Gravis

Clinical efficacy and cost per improved outcome of treatments for generalized myasthenia gravis: evidence from a network meta-analysis and cost-per-responder analysis. (PubMed, Curr Med Res Opin) - "A de novo NMA was performed using data from Phase III placebo-controlled trials of efgartigimod IV, inebilizumab, nipocalimab, ravulizumab, rozanolixizumab, and zilucoplan, including recently approved therapies. Efgartigimod IV had the lowest CPIO across all QMG and MG-ADL thresholds, significantly lower than almost all other treatments (p < 0.05). Among approved effective biologic therapies for gMG, efgartigimod IV offers a favorable combination of clinical efficacy and economic value."

HEOR • Journal • Retrospective data • CNS Disorders • Myasthenia Gravis

Nipocalimab Effect On Immunoglobulin G Subclasses In Patients With Generalized Myasthenia Gravis (CNSF 2026) - "Nipocalimab significantly inhibits FcRn-mediated IgG recycling, leading to rapid, greater and sustained reductions in all IgG subclasses and pathogenic anti-AChR autoantibodies in gMG compared with efgartigimod and rozanolixizumab, highlighting its therapeutic potential."

Clinical • CNS Disorders • Myasthenia Gravis

Corticosteroid dose tapering during treatment with rozanolixizumab in patients with generalised myasthenia gravis: Post hoc analysis (CNSF 2026) - P3 | "Over a third of patients decreased or discontinued corticosteroids during long-term rozanolixizumab treatment, without protocol guidance on corticosteroid tapering."

Clinical • Retrospective data • CNS Disorders • Myasthenia Gravis

Repeated rozanolixizumab cycles showed consistent benefit in MuSK Ab+ gMG (UCB) (UCB Press Release) - "Final pooled data from the Phase 3 MycarinG study and its open-label extension studies showed consistent improvements from baseline in MG-ADL and QMG scores across adult patients with MuSK antibody-positive (MuSK Ab+) gMG."

Retrospective data • Immunology • Myasthenia Gravis

FcRn-Targeted Alpha Therapy Using [225Ac]Ac-Macropa-Rozanolixizumab Enables Potent Antitumor Activity. (PubMed, Mol Imaging Biol) - "[225Ac]Ac-Macropa-rozanolixizumab demonstrated FcRn-specific α-particle cytotoxicity and favorable dosimetric properties in a proof-of-concept xenograft model. These findings support FcRn as a potential target for radioimmunotherapy. Further studies are required to evaluate toxicity, endogenous IgG competition and translational applicability."

IO biomarker • Journal • Hematological Disorders • Hematological Malignancies • Oncology • Solid Tumor

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

Long-term rozanolixizumab treatment cycles showed repeated and consistent benefit across ocular and wider generalized myasthenia gravis (gMG) symptoms (UCB) (UCB Press Release) - "Final pooled data from the Phase 3 MycarinG study and its open-label extension studies showed improvements across ocular signs and symptoms, alongside consistent benefits in bulbar, respiratory and limb weakness/gross motor function in adults living with gMG compared with placebo."

Retrospective data • Immunology • Myasthenia Gravis

Retraction: Aseptic Meningitis Following Rozanolixizumab in a Patient With Refractory Anti-MuSK Positive Myasthenia Gravis: A Case Report. (PubMed, Neurohospitalist) - "[This retracts the article DOI: 10.1177/19418744251378584.]."

Journal • CNS Disorders • Infectious Disease • Myasthenia Gravis

What We Have Learnt From Comparative Analyses? (ICNMD 2026) - "During the last decade, considerable advances have been made in research and approval of targeted therapies such as complement or neonatal Fc (FcRn) inhibitors which have now entered clinical practice. Literature review and experience from real world use of targeted therapies in MG Clinical experience started with eculizumab for refractory MG and efgartigimod, followed by the approval or ravulizumab and zilucoplan as complement inhibitors, and rozanolixizumab and nipocalimab as FcRn inhibitors. The evidence to guide the choice between anti-complement or anti-FcRn inhibition in severe MG deterioration is still limited. Apart from issues related to autoantibody specificities (anti-acetylcholine vs MuSK), decisions must be made taking into account each patient previous history (particulary the frequency of relapses and need for rescue therapy), comorbidities, and risk of infection, as well as the need to set a strategy as a form of "bridging therapy" or as a..."

CNS Disorders • Immunology • Infectious Disease • Myasthenia Gravis • Respiratory Diseases • Septic Shock

Repeated Cycles of an FcRn‑Targeting Therapy in Antibody‑Positive Generalised Myasthenia Gravis (ICNMD 2026) - P3 | "Patients with MuSK Ab+ gMG who received rozanolixizumab treatment consistently had improvements from baseline in MG-ADL and QMG scores across cycles, with an acceptable safety profile."

CNS Disorders • Myasthenia Gravis

Early First-Week Changes in MG-ADL and MG-QOL-15 During C5 and FcRn-Inhibitors Treatment in Myasthenia Gravis (ICNMD 2026) - "The aim of this study was to assess early daily variations in functional status and quality of life during the first week of treatment with complement (C5) and neonatal Fc receptor (FcRn) inhibitors in patients with gMG. We conducted a retrospective multicenter analysis of 95 patients with gMG receiving complement inhibitors (eculizumab, ravulizumab, zilucoplan) or FcRn inhibitors (efgartigimod, rozanolixizumab). Both C5 and FcRn inhibitors are associated with rapid and clinically meaningful improvements in daily functioning and quality of life within the first week of treatment in gMG. Improvements are detectable as early as day 3 and appear comparable across therapeutic classes, underscoring the fast onset of action of these innovative immunotherapies"

CNS Disorders • Myasthenia Gravis

The Uptake, Intracellular Trafficking and Recycling of FcRn-Blocking Therapeutics in Human Endothelial Cells in Vitro (ICNMD 2026) - "These data demonstrate the impact of different structural features of FcRn inhibitors on functional outcomes on cells in vitro. Further studies are needed to understand the direct clinical implications of such data in gMG and other IgG autoantibody-driven disorders. However, these different characteristics may in part drive the observed pharmacodynamic differences in gMG patients, that result in different requirements in terms of dosing."

Preclinical • CNS Disorders • Myasthenia Gravis

Quality of Life in Myasthenia Gravis: Post Hoc Pooled Analysis of Phase 3 Rozanolixizumab Studies (ICNMD 2026) - P3 | "Long-term rozanolixizumab treatment cycles led to improvements in all MG-QoL15r item-level scores, demonstrating consistent efficacy across the range of impacts on health-related QoL that MG symptoms have. Funding: UCB."

HEOR • P3 data • Retrospective data • CNS Disorders • Immunology • Myasthenia Gravis

Minimal Symptom Expression in Myasthenia Gravis: A Post Hoc Analysis of Phase 3 Rozanolixizumab Studies (ICNMD 2026) - P3 | "For patients with gMG who achieved MSE in Cycle 1, within each subsequent cycle, the majority achieved MSE, suggesting consistent efficacy with long-term rozanolixizumab treatment. Funding: UCB."

P3 data • Retrospective data • CNS Disorders • Myasthenia Gravis

Effect of Rozanolixizumab on MG-ADL and QMG Items: A Post-Hoc Phase 3 Final Pooled Analysis (ICNMD 2026) - P3 | "Long-term rozanolixizumab treatment cycles led to improvements from baseline across ocular, bulbar, respiratory and limb weakness/gross motor items in MG-specific outcomes, demonstrating consistent benefit across a broad range of signs and symptoms. Funding: UCB."

P3 data • Retrospective data • CNS Disorders • Myasthenia Gravis

Effect of Rozanolixizumab on Ocular Symptoms in Generalised Myasthenia Gravis: Analysis of Phase 3 Studies (ICNMD 2026) - P3 | "Long-term rozanolixizumab treatment cycles led to improvements across ocular items in MG-specific outcome measures, suggesting a benefit for patients with gMG experiencing ocular signs and symptoms. Funding: UCB."

P3 data • CNS Disorders • Myasthenia Gravis

Characterisation of Patients With Generalised Myasthenia Gravis on Rozanolixizumab Treatment in European Managed Access Programmes (ICNMD 2026) - P3 | "The baseline characteristics of the patients who entered the rozanolixizumab MAPs were indicative of a population with moderate-to-severe gMG. Funding: UCB."

Clinical • CNS Disorders • Immunology • Myasthenia Gravis