Zilbrysq (zilucoplan) / UCB - LARVOL DELTA

Letter to editor: Early real-life experience on zilucoplan for generalized myasthenia gravis: ZILU25 multicenter observational study. (PubMed, J Neurol Sci) - No abstract available

Journal • Observational data • CNS Disorders • Myasthenia Gravis

Assessing the Value Contribution of Vyvgart® (Efgartigimod Alfa) in the Treatment of Generalized Myasthenia Gravis with Acetylcholine Receptor Antibody in Spain Through Multi-criteria Decision Analysis. (PubMed, Adv Ther) - "Efgartigimod has been recognised as a valuable option for gMG AChR+ treatment in Spain by a multidisciplinary panel of experts through the application of MCDA, receiving higher scores compared with ravulizumab, zilucoplan, and rozanolixizumab."

Journal • CNS Disorders • Immunology • Myasthenia Gravis • Rare Diseases

Letter to the editor: Early real-life experience on Zilucoplan for generalized myasthenia gravis: ZILU25 multicenter observational study. (PubMed, J Neurol Sci) - No abstract available

Journal • Observational data • CNS Disorders • Myasthenia Gravis

Authors' reply to comment letter on ZILU25 study. (PubMed, J Neurol Sci) - No abstract available

Journal • CNS Disorders • Myasthenia Gravis

Comments on ZILU25: Real-world efficacy of Zilucoplan in generalized myasthenia gravis. (PubMed, J Neurol Sci) - No abstract available

Journal • Real-world evidence • CNS Disorders • Myasthenia Gravis

Novel Therapies for Generalized Myasthenia Gravis: Insights Into FcRn and Complement Inhibition. (PubMed, Am J Ther) - "These therapies offer personalized, effective options for MG management, though challenges in cost and access remain. Future research should address long-term outcomes and biomarkers."

Journal • Review • CNS Disorders • Immunology • Infectious Disease • Myasthenia Gravis

HEALEY ALS Platform Trial - Master Protocol (clinicaltrials.gov) - P2/3 | N=1500 | Active, not recruiting | Sponsor: Merit E. Cudkowicz, MD | Trial completion date: Apr 2026 ➔ Aug 2028 | Trial primary completion date: Jul 2025 ➔ Nov 2027

Trial completion date • Trial primary completion date • Amyotrophic Lateral Sclerosis • CNS Disorders

Body weight distribution of US patients with myasthenia gravis. (PubMed, J Med Econ) - "The dosing requirements of certain therapies (efgartigimod intravenous, nipocalimab, ravulizumab, rozanolixizumab, zilucoplan) are based on patient's body weight. The annual cost of health-care-provider-administered treatment for patients with gMG was estimated at $223,272-$648,960 depending on drug and body weight; annual cost was highest for patients who weighed >80 kg (up to $648,960 versus up to $467,044 for patients who weighed <80 kg). Our findings demonstrate that patients with MG may be heavier on average than previously assumed, which may influence the cost-effectiveness of gMG therapies with weight-based dosing."

Journal • CNS Disorders • Immunology • Myasthenia Gravis

Cellular and humoral vaccination response under immunotherapies-German consensus on vaccination strategies in neurological autoimmune diseases. (PubMed, Ther Adv Neurol Disord) - "The specific humoral and cellular response to vaccination can be compromised under alemtuzumab, azathioprine, cladribine, cyclophosphamide, CD19/CD20 antibodies (inebilizumab, ocrelizumab, ofatumumab, rituximab, ublituximab), dimethyl fumarate/diroximel fumarate, FcRn inhibitors (efgartigimod, rozanolixizumab), complement C5 inhibitors (eculizumab, ravulizumab, zilucoplan), interleukin-6 receptor antibodies (tocilizumab, satralizumab), intravenous immunoglobulins, long-term steroid administration, methotrexate, mitoxantrone, mycophenolate mofetil, tacrolimus, teriflunomide, tumor necrosis factor-α blockers, and sphingosine-1-phosphate receptor modulators (fingolimod, ozanimod, ponesimod, siponimod), as well as after autologous stem cell transplantation...However, the humoral and cellular vaccination response may be impaired under immunotherapy necessitating close monitoring. Here, we provide applicable recommendations to optimize immunization for individuals receiving..."

Journal • CNS Disorders • Immunology • Infectious Disease • Oncology • Transplantation • IL6R

Human in vitro neuromuscular junction model to functionally dissect the pathogenic mechanism of anti-AChR autoantibody-positive myasthenia gravis. (PubMed, BMC Pharmacol Toxicol) - "These findings offer a mechanistic rationale for the observed clinical response in patients with anti-AChR autoantibody-positive myasthenia gravis treated with C5 inhibitors. The in vitro neuromuscular junction model provides a robust platform for studying the mechanistic pathways of complement-mediated damage and evaluating therapeutic interventions for myasthenia gravis."

Journal • Preclinical • CNS Disorders • Immunology • Myasthenia Gravis

Real-world data on breakthrough hemolysis in patients with paroxysmal nocturnal hemoglobinuria treated with proximal and terminal complement inhibitors. (ASH 2025) - "Four patients were treated with factor D inhibitor danicopan(n=3) or iptacopan (n=1) concurrently with ravulizumab.A total of 49 BTH events were identified; while on eculizumab (26 events, 12 patients), ravulizumab (14events, 7 patients), ravulizumab and danicopan (6 events, 3 patients), pegcetacoplan (1 event, 1 patient),zilucoplan (2 events, 2 patients), and no events while on iptacopan. Twelve BTH events were incidentally detected during routine CIadministration; more frequent lab draws may be beneficial for patients with chronic hemolysis. Moredata is needed to understand the BTH rates of different CIs and guide clinicians in BTH management."

Clinical • Real-world • Real-world evidence • Anemia • Aplastic Anemia • Breast Cancer • Complement-mediated Rare Disorders • Infectious Disease • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Solid Tumor • CD55 • CD59 • HP

Discovery of Zilucoplan: A Complement C5 Inhibitor for Treatment of Anti-Acetylcholine Receptor (AChR) Antibody-Positive Generalized Myasthenia Gravis (gMG). (PubMed, J Med Chem) - "In Ribosome Display and Related Technologies: Methods and Protocols; Douthwaite, J. A., Jackson, R. H., Eds.; Springer: New York, 2012; pp 367-390.) against C5 and approved for the treatment of gMG. We highlight the key steps and rationale behind the peptide medicinal chemistry optimization of the initial screening hits, that led to improved potency, stability, and pharmacokinetic properties."

Journal • CNS Disorders • Myasthenia Gravis

Effectiveness and Safety of Zilucoplan Treatment for French Patients With Myasthenia Gravis Over 3 Months (clinicaltrials.gov) - P=N/A | N=55 | Recruiting | Sponsor: Centre Hospitalier Universitaire de Nice | Not yet recruiting ➔ Recruiting | Trial primary completion date: Nov 2025 ➔ Feb 2026

Enrollment open • Trial primary completion date • CNS Disorders • Myasthenia Gravis

Early real-life experience on Zilucoplan for generalized myasthenia gravis: ZILU25 multicenter observational study. (PubMed, J Neurol Sci) - "Zilucoplan was well-tolerated and effective in most patients with AChR-positive gMG. A clinical meaningful effect was reported since the first week and was sustained after 24 weeks. Further studies are needed to confirm these preliminary real-life data."

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

Complement Inhibitors in Generalized Myasthenia Gravis: Comparison of Administration Schedules, Efficacy, and Safety. (PubMed, J Clin Med) - "Background: Eculizumab, Ravulizumab, and Zilucoplan are inhibitors of terminal complement protein C5 (C5IT) approved for the treatment of generalized Myasthenia Gravis (gMG). Most of the reported adverse events were mild to moderate; the more severe events included one case of Stevens-Johnson syndrome (Ravulizumab) and episodes of pneumonia (Eculizumab, Ravulizumab). The comparison of C5IT did not bring out significant differences in terms of clinical efficacy and safety, representing a valid therapeutic option when traditional therapies fail to control disease symptoms."

Journal • CNS Disorders • Infectious Disease • Myasthenia Gravis • Pneumonia • Respiratory Diseases • Steven-Johnson Syndrome

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

Costs and Number Needed to Treat (NNT) to Achieve Improvements With C5 Inhibitors in Generalized Myasthenia Gravis From the Japanese Healthcare Perspective: A Network Meta-Analysis (ISPOR-EU 2025) - P3 | "Zilucoplan showed similar or lower NNTs (0.31-0.48) and CPIO (M¥10.68-M¥16.41) compared to ravulizumab (NNT: 0.50-0.59; CPIO: M¥23.61-M¥27.83) and eculizumab (NNT: 0.30-0.52; CPIO: M¥19.32-M¥33.68) across all CFB endpoints. Furthermore, zilucoplan demonstrated superior cost-benefit, requiring lower treatment costs to achieve each improved outcome."

Retrospective data • CNS Disorders • 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

Zilucoplan for Severe gMG Exacerbations (clinicaltrials.gov) - P3 | N=15 | Not yet recruiting | Sponsor: Miriam Freimer

New P3 trial • CNS Disorders • Myasthenia Gravis

SUSTAINED MINIMAL SYMPTOM EXPRESSION IN GENERALISED MYASTHENIA GRAVIS: A 120-WEEK POST HOC ANALYIS OF RAISE-XT (WCN 2025) - P2, P3 | "Zilucoplan demonstrated sustained efficacy, as shown by maintenance of MSE response up to 120 weeks of treatment."

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

Manifestation of morphea in a patient with myasthenia gravis under therapy with zilucoplan. (PubMed, J Dtsch Dermatol Ges) - No abstract available

Journal • CNS Disorders • Myasthenia Gravis

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