Real-world effectiveness and safety of zilucoplan in patients with anti-AChR myasthenia gravis: A retrospective cohort study in France

Objectives To evaluate the effectiveness and safety of zilucoplan in the real-world treatment setting in France. Methods This retrospective cohort study evaluated patients with generalized anti-AChR myasthenia gravis (MG) failing current therapy enrolled in the French early access program (EAP) for zilucoplan. Patients were evaluated at enrollment and at Months 1, 3 and 6 with the MG-ADL score, the Myasthenic Muscle Score (MMS-Garches), and the MG-quality of life questionnaire (MG-QoL15r). MG crises and on-treatment adverse events were documented. MG medication use was compared between the six months before and after enrollment. Results Forty-eight patients were enrolled and treated with zilucoplan (mean age: 56.2 years; 50.9% men). Thirty patients achieved six months follow-up before the EAP ended. Three patients discontinued zilucoplan due to lack of efficacy. Mean MG-ADL score decreased from 7.4 ± 4.4 to M0 to 2.5 ± 2.7 at M6 ( p < 0.0001). 9/43 patients achieved minimal symptom expression at M3 and 12/30 at M6. Mean MMS-Garches score was 67.5 ± 20.0 at M0 and 87.5 ± 13.2 at M6 ( p < 0.0001). Mean MG-QoL15r score was 18.2 ± 8.6 at M0 and 8.7 ± 8.3 at M6 ( p = 0.0156). 15 patients experienced adverse events, leading to discontinuation in four cases. The mean oral corticosteroid dose decreased from 28.8 ± 11.8 mg to 17.9 ± 11.8 ( p = 0.001). Six patients experienced MG crises on treatment, compared to 22 in the six months preceding enrollment. treatment. Discussion Following initiation of zilucoplan treatment, we observed an improvement in MG symptoms and a reduction in MG crises and in exposure to oral corticosteroids. Overall, zilucoplan was well-tolerated with no major adverse events reported. Trial registration information The study was registered in the clinicaltrials.gov trial registry under the number NCT06815133 ( https://clinicaltrials.gov/study/NCT06815133 ) on February 4 th , 2025.

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Journal
Journal of Neuromuscular Diseases
Published
2026-03-29
DOI
https://doi.org/10.1177/22143602261437792
Primary Topic
Myasthenia Gravis and Thymoma
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article
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article

Real-world effectiveness and safety of zilucoplan in patients with anti-AChR myasthenia gravis: A retrospective cohort study in France

S. Beltran, A. Behin, Vincent Fabry, Julien Praline et al.
Journal of Neuromuscular Diseases
Myasthenia Gravis and Thymoma
article

Real-world effectiveness and safety of zilucoplan in patients with anti-AChR myasthenia gravis: A retrospective cohort study in France

S. Beltran, A. Behin, Vincent Fabry, Julien Praline, Diane Friedman, Guillaume Taïeb, Luce Barbat du Closel, Clémence Marois, Antoine Pégat, É. Lagrange, P. Merle, Juliette Svahn, Simon Frachet, Giulia Tammam, Blandine Acket, Fanny Duval, Shahram Attarian, Pascal Cintas, Michele Cavalli, Emmanuelle Salort-Campana, Abderhmane Slioui, Aleksandra Nadaj Pakleza, Angela Puma, Saskia Bresch, Luisa Villa, Jean-Baptiste Noury, Guilhem Sole, Djillali Annane, Françoise Bouhour, Sabrina Sacconi, Céline Tard
article en

Abstract

Objectives To evaluate the effectiveness and safety of zilucoplan in the real-world treatment setting in France. Methods This retrospective cohort study evaluated patients with generalized anti-AChR myasthenia gravis (MG) failing current therapy enrolled in the French early access program (EAP) for zilucoplan. Patients were evaluated at enrollment and at Months 1, 3 and 6 with the MG-ADL score, the Myasthenic Muscle Score (MMS-Garches), and the MG-quality of life questionnaire (MG-QoL15r). MG crises and on-treatment adverse events were documented. MG medication use was compared between the six months before and after enrollment. Results Forty-eight patients were enrolled and treated with zilucoplan (mean age: 56.2 years; 50.9% men). Thirty patients achieved six months follow-up before the EAP ended. Three patients discontinued zilucoplan due to lack of efficacy. Mean MG-ADL score decreased from 7.4 ± 4.4 to M0 to 2.5 ± 2.7 at M6 ( p < 0.0001). 9/43 patients achieved minimal symptom expression at M3 and 12/30 at M6. Mean MMS-Garches score was 67.5 ± 20.0 at M0 and 87.5 ± 13.2 at M6 ( p < 0.0001). Mean MG-QoL15r score was 18.2 ± 8.6 at M0 and 8.7 ± 8.3 at M6 ( p = 0.0156). 15 patients experienced adverse events, leading to discontinuation in four cases. The mean oral corticosteroid dose decreased from 28.8 ± 11.8 mg to 17.9 ± 11.8 ( p = 0.001). Six patients experienced MG crises on treatment, compared to 22 in the six months preceding enrollment. treatment. Discussion Following initiation of zilucoplan treatment, we observed an improvement in MG symptoms and a reduction in MG crises and in exposure to oral corticosteroids. Overall, zilucoplan was well-tolerated with no major adverse events reported. Trial registration information The study was registered in the clinicaltrials.gov trial registry under the number NCT06815133 ( https://clinicaltrials.gov/study/NCT06815133 ) on February 4 th , 2025.

Journal of Neuromuscular Diseases
Université d'Orléans (FR), Centre National de la Recherche Scientifique (FR), Inserm (FR), Université de Bretagne Occidentale (FR), Université de Versailles Saint-Quentin-en-Yvelines (FR), Université de Lille (FR), Centre Hospitalier Universitaire de Grenoble (FR), Sorbonne Université (FR), Centre Hospitalier Universitaire Amiens-Picardie (FR), Hôpital Bretonneau (FR), Hospices Civils de Lyon (FR), Hôpital Pierre Wertheimer (FR), Hôpital Pellegrin (FR), Institut de Recherche sur le Cancer et le Vieillissement de Nice (FR), Pitié-Salpêtrière Hospital (FR), Centre Hospitalier Régional Universitaire de Brest (FR), Hôpital Purpan (FR), Hôpital Pasteur (FR), Centre hospitalier universitaire d'Orléans (FR), Hôpital de la Timone (FR), Laboratoire Médiations (FR), Université de Limoges (FR), Université de Strasbourg (FR), Université Grenoble Alpes (FR)
Openalex Percentile: Top 4%
Myasthenia Gravis and Thymoma
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