Zilbrysq (zilucoplan) / UCB - LARVOL DELTA

Zilucoplan Shows Rapid, Durable, Steroid-Sparing Benefits in gMG (AJMC) - "Zilucoplan (Zilbrysq; UCB), a once-daily subcutaneous C5 complement inhibitor, continues to demonstrate sustained clinical value in patients with acetylcholine receptor antibody–positive (AChR Ab+) generalized myasthenia gravis (gMG)....Overall, 61.1% of patients with baseline and week 120 data available were able to reduce their steroid dose or discontinue their steroid use compared with 9.3% of all patients (N = 86) who had to increase from their initial dose by a mean of 11.6 mg or start on the treatment. In those who reduced their dose, the mean reduction was 15.5 mg—from 23.0 mg/day at baseline to 7.5 mg/day by week 20—as they demonstrated a mean (SD) 6.6 (3.6)-point improvement on their MG-ADL score, 'supporting that symptom control was not compromised by steroid tapering,' the authors wrote."

Retrospective data • Myasthenia Gravis

Changes in corticosteroid and non-steroidal immunosuppressive therapy with long-term zilucoplan treatment in generalized myasthenia gravis. (PubMed, J Neurol) - P2, P3 | "Treatment with zilucoplan allowed for reduction or discontinuation of corticosteroids in the majority of patients and NSIST in about a third of patients, while maintaining efficacy."

Clinical • Journal • CNS Disorders • Myasthenia Gravis

Early and sustained response over time with zilucoplan in generalised Myasthenia Gravis: 120-week post hoc analysis of RAISE-XT (CNSF 2025) - No abstract available

Retrospective data • CNS Disorders • Myasthenia Gravis

Efficacy of zilucoplan in patients with generalised myasthenia gravis who have not previously received immunoglobulin or plasma exchange: A subgroup analysis from the Phase 3 RAISE study. (PubMed, J Neurol Sci) - P3 | "In the RAISE study, patients without prior immunoglobulin/PLEX, who had milder disease, experienced improvements in MG-specific outcomes with zilucoplan that were comparable to those seen in patients with prior immunoglobulin/PLEX. These findings may support early use of zilucoplan in the gMG treatment paradigm."

Journal • P3 data • CNS Disorders • Myasthenia Gravis

The Role of Complement in the Pathogenesis and Treatment of Myasthenia Gravis. (PubMed, Cells) - "This has led to the development of the first C5 inhibitors approved for myasthenia gravis with AchR antibodies: eculizumab, ravulizumab, and zilucoplan. Other clinical trials are currently in progress, investigating the potential therapeutic role of other targets, including the Factor B inhibition or hepatic synthesis of the C5 protein. Other proposed potential targets that have not yet been clinically tested are also discussed in this review article."

Journal • Review • CNS Disorders • Immunology • Myasthenia Gravis

Long-term safety and efficacy of zilucoplan in gMG: 120-week interim analysis of RAISE-XT [Encore Abstract] (JSNE 2025) - No abstract available

Clinical

A Study to Assess Zilucoplan Concentration in Breast Milk of Healthy Lactating Women (clinicaltrials.gov) - P1 | N=15 | Not yet recruiting | Sponsor: UCB Biopharma SRL

New P1 trial

Switching to Subcutaneous Zilucoplan from Intravenous Complement Component 5 Inhibitors in Myasthenia Gravis: Patient Preference and Satisfaction from a Phase 3b Study (P1-11.009). (PubMed, Neurology) - P3 | "Twenty-six patients enrolled and received zilucoplan; 16 switched from eculizumab and 10 from ravulizumab. Howard has a non-compensated relationship as a Committee member with American Assoc Neuromuscular and Electrodiagnostic Medicine that is relevant to AAN interests or activities. Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff."

Journal • P3 data • CNS Disorders • Myasthenia Gravis

Effect of Zilucoplan on Myasthenia Gravis-Specific Outcome Subdomain Scores in RAISE: A Phase 3 Study (S34.004). (PubMed, Neurology) - P3 | "Howard has a non-compensated relationship as a Committee member with American Assoc Neuromuscular and Electrodiagnostic Medicine that is relevant to AAN interests or activities. Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff."

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

Zilucoplan for Successful Early Weaning From Mechanical Ventilation and Avoiding Tracheostomy in an 85-Year-Old Woman Experiencing Myasthenic Crisis: A Case Report. (PubMed, Cureus) - "Initial treatment with Intravenous immunoglobulin and intravenous methylprednisolone showed limited efficacy, making weaning from mechanical ventilation challenging. This case supports the early addition of zilucoplan with conventional rescue therapies for myasthenic crisis may have enabled earlier extubation and the avoidance of tracheostomy. Further studies are needed to determine the universal applicability of add-on therapies in myasthenic crisis, considering comorbidities and complications."

Journal • Cardiovascular • CNS Disorders • Congestive Heart Failure • Diabetes • Heart Failure • Metabolic Disorders • Myasthenia Gravis • Respiratory Diseases

UCB presents latest research across leading neurology portfolio at American Academy of Neurology (AAN) meeting 2025 (PRNewswire) - "24 scientific abstracts reflect UCB's ongoing commitment to improving outcomes for people living with neurological conditions, including rare epilepsies Dravet syndrome and Lennox-Gastaut syndrome, as well as generalized myasthenia gravis and thymidine kinase 2 deficiency; Research includes disease course data of untreated patients with thymidine kinase 2 deficiency from largest international dataset; Also features new data on generalized myasthenia gravis treatments RYSTIGGO (rozanolixizumab-noli) and ZILBRYSQ (zilucoplan) including an open-label extension study investigating rozanolixizumab self-administration and a phase 3 study on the effect of zilucoplan on specific outcome scores; Data on FINTEPLA (fenfluramine), BRIVIACT (brivaracetam) CV, and investigational therapy STACCATO alprazolam showcase commitment to people living with epilepsies and their unmet needs."

Clinical data • Epilepsy • Immunology • Myasthenia Gravis

Switching to Subcutaneous Zilucoplan from Intravenous Complement Component 5 Inhibitors in Myasthenia Gravis: Patient Preference and Satisfaction from a Phase 3b Study (AAN 2025) - P3 | "Treatment satisfaction was measured using the 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9; scored 0–100; exploratory endpoint). Twenty-six patients enrolled and received zilucoplan; 16 switched from eculizumab and 10 from ravulizumab. Zilucoplan demonstrated a favorable safety profile. More patients preferred subcutaneous to intravenous treatment, and overall treatment satisfaction increased after switching from intravenous C5 inhibitors to subcutaneous zilucoplan."

Clinical • P3 data • CNS Disorders • Myasthenia Gravis

Evaluating the Comparative Effectiveness of Emerging Immunomodulatory Therapies for Patients with Generalized Myasthenia Gravis (AAN 2025) - "Comparative evaluations of these treatments are necessary for guiding informed clinical decision-making.Design/A Bayesian network meta-analysis NMA was conducted to compare the efficacy of neonatal Fc receptor inhibitors (efgartigimod intravenous [IV], rozanolixizumab) and complement inhibitors (ravulizumab, zilucoplan) versus placebo. Fc receptor inhibitors, particularly efgartigimod IV, show a more favorable efficacy profile compared to ravulizumab and zilucoplan. Further results, including data on nipocalimab and inebilizumab, will be presented in updates to the analysis."

Clinical • HEOR • Immunomodulating • CNS Disorders • Myasthenia Gravis

C5-Complement Inhibitors Administration for Treatment of Generalized Myasthenia Gravis: A Systematic Review and Meta-analysis (AAN 2025) - "Objective:To evaluate the efficacy of complement inhibitors for treating myasthenia gravis.Background:C5 inhibitors, including eculizumab, zilucoplan, and ravulizumab, have been considered to be beneficial for patients with myasthenia gravis, acting by blocking the formation of the membrane attack complex, consequently reducing damage to neuromuscular junction cells and protecting against degradation of acetylcholine receptors. Our meta-analysis demonstrates that complement inhibitors exhibited greater efficacy than placebo across multiple scores of myasthenia gravis treatment. Furthermore, they did not present significant side effects."

Retrospective data • Review • CNS Disorders • Myasthenia Gravis • Pain

Effect of Zilucoplan on Myasthenia Gravis–Specific Outcome Subdomain Scores in RAISE: A Phase 3 Study (AAN 2025) - P3 | "Zilucoplan treatment led to improvements relative to placebo across all subdomain scores in MG-ADL and QMG."

P3 data • CNS Disorders • Myasthenia Gravis

Subcutaneous zilucoplan: evaluation of reproductive toxicology. (PubMed, Reprod Toxicol) - "These reproductive toxicity studies demonstrate no adverse effect of zilucoplan on male fertility or maternal or embryo-foetal outcomes, and no pre- or postnatal toxicity in NHPs receiving daily zilucoplan. Data from the ex vivo placental transfer model and the lack of effect of FcRn-mediated transfer on zilucoplan suggest that placental transfer is low."

Journal • CNS Disorders • Myasthenia Gravis

On Growth Path for a Decade plus: Strong Launch Execution driving Company Growth (PRNewswire) - "Net sales were up by 15% to € 5.61 billion (+17% CER1) driven by a strong, triple- and double-digit growth performance of newly launched growth drivers: BIMZELX, EVENITY, FINTEPLA, RYSTIGGO and ZILBRYSQ as well as solid contribution from CIMZIA and BRIVIACT reaching its peak sales two years ahead of target...EVENITY (romosozumab) since launch globally reached more than 900 000 (2023: 600 000) women living with postmenopausal osteoporosis at high risk of fracture. Net sales in Europe increased by 71% to € 103 million (+71% CER) after € 60 million in 2023...BRIVIACT (brivaracetam) was used by over 232 000 people living with epilepsy and increased net sales to € 686 million, achieving its peak sales target of 'at least € 600 million' well before 2026."

Sales • Sales projection • Ankylosing Spondylitis • Crohn's disease • Epilepsy • Hidradenitis Suppurativa • Inflammatory Arthritis • Myasthenia Gravis • Osteoporosis • Psoriasis • Psoriatic Arthritis • Pustular Psoriasis • Rheumatology • Spondylarthritis

Ravulizumab and other complement inhibitors for the treatment of autoimmune disorders. (PubMed, Mult Scler Relat Disord) - "Based on the importance of intermediate and terminal components of the complement cascade during disease pathogenesis, in the last few years, a mAb targeting the C5 complement factor (anti-C5, eculizumab) has already been in use for treating AQP4-IgG positive NMOSD. Here, we discuss the main findings obtained during the phase 3 clinical trial (CHAMPIONNMOSD [NCT0420126]) for ravulizumab and new developments in anti-complement therapy, with other complement inhibitors for the treatment of autoimmune diseases and paroxysmal nocturnal hemoglobinuria (PNH) (zilucoplan, avacopan and, pegcetacoplan). The approval of the new long-acting anti-C5 mAb adds another therapeutic option to the already existing inhibitors of complement currently in use, increasing therapeutic options for inflammatory and autoimmune diseases."

Journal • Review • CNS Disorders • Complement-mediated Rare Disorders • Hematological Disorders • Immunology • Neuromyelitis Optica Spectrum Disorder • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Recommendations [Google translation] (Danish Medicines Council) - "The Danish Medical Council does not recommend zilucoplan as an adjunct to standard treatment for patients with the rare autoimmune disease generalized myasthenia gravis who are seropositive for antibodies directed against the acetylcholine receptor....The Danish Medicines Council does not recommend sotatercept for the treatment of pulmonary arterial hypertension (high blood pressure in the pulmonary circulation) in adult patients in functional class II and III."

Reimbursement • Myasthenia Gravis • Pulmonary Arterial Hypertension

A Study to Evaluate the Safe and Effective Use of a Zilucoplan Auto-injector by Study Participants With Generalized Myasthenia Gravis (clinicaltrials.gov) - P3 | N=31 | Completed | Sponsor: UCB Biopharma SRL | Active, not recruiting ➔ Completed

Trial completion • CNS Disorders • Myasthenia Gravis

Efficacy and Safety of Zilucoplan in Amyotrophic Lateral Sclerosis: A Randomized Clinical Trial. (PubMed, JAMA Netw Open) - P2/3 | "Eligible participants were randomized in a 3:1 ratio to receive zilucoplan or matching placebo within strata of edaravone and/or riluzole use for a planned duration of 24 weeks. The adaptive platform design of the HEALEY ALS Platform Trial made it possible to test a new investigational product with efficient use of time and resources. ClinicalTrials.gov Identifier: NCT04297683."

Clinical • Journal • Amyotrophic Lateral Sclerosis • CNS Disorders • Inflammation

Effectiveness and Safety of Zilucoplan Treatment for French Patients with Myasthenia Gravis Over 3 Months (clinicaltrials.gov) - P=N/A | N=55 | Not yet recruiting | Sponsor: Centre Hospitalier Universitaire de Nice

New trial • CNS Disorders • Myasthenia Gravis

A Study to Evaluate the Safe and Effective Use of a Zilucoplan Auto-injector by Study Participants With Generalized Myasthenia Gravis (clinicaltrials.gov) - P3 | N=31 | Active, not recruiting | Sponsor: UCB Biopharma SRL | Recruiting ➔ Active, not recruiting

Enrollment closed • CNS Disorders • Myasthenia Gravis

Advances in Disease-Modifying Therapeutics for Chronic Neuromuscular Disorders. (PubMed, Semin Respir Crit Care Med) - "For myasthenia gravis (MG), efgartigimod, ravulizumab, rozanolixizumab, and zilucoplan have been Food and Drug Administration (FDA)-approved for the treatment of acetylcholine receptor (AChR) antibody-positive generalized MG in the past 2 years...For spinal muscular atrophy (SMA), nusinersen (intrathecal route) and risdiplam (oral route) modify the splicing of the SMN2 gene, increasing the production of normal survival motor neuron (SMN) protein...For late-onset Pompe disease (LOPD), avalglucosidase alfa has shown a greater improvement in respiratory function, ambulation, and functional outcomes in comparison to alglucosidase alfa, and cipaglucosidase alfa combined with miglustat has shown improvement in respiratory and motor function in a cohort of enzyme replacement therapy-experienced LOPD patients. Amyotrophic lateral sclerosis (ALS) remains a challenge. The two most recent FDA-approved medications, namely sodium phenylbutyrate and tofersen, may slow down the disease..."

Journal • Amyotrophic Lateral Sclerosis • CNS Disorders • Genetic Disorders • Movement Disorders • Myasthenia Gravis • Pompe Disease • Rare Diseases • SMN2

A Study to Evaluate Zilucoplan Injected Subcutaneously Either by a Prefilled Syringe or an Auto-injector in Healthy Adult Participants (clinicaltrials.gov) - P1 | N=14 | Completed | Sponsor: UCB Biopharma SRL | Active, not recruiting ➔ Completed

Trial completion