Safety and efficacy of chronic weekly rozanolixizumab in generalized myasthenia gravis: the randomized open-label extension MG0004 study

Authors

Vera Bril, Ellen and Martin Prosserman Centre for Neuromuscular Diseases, Toronto General Hospital, University of Toronto, Toronto, Ontario, Canada. vera.bril@utoronto.ca.
Artur Drużdż, Department of Neurology, Municipal Hospital, Poznań, Poland.
Julian Grosskreutz, Precision Neurology of Neuromuscular Diseases, Department of Neurology, University of Lübeck, Lübeck, Germany.
Ali A. Habib, MDA ALS and Neuromuscular Center, Department of Neurology, University of California, Irvine, Orange, CA, USA.
Henry J. Kaminski, Department of Neurology and Rehabilitation Medicine, George Washington University, Washington, DC, USA.
Renato Mantegazza, Department of Neuroimmunology and Neuromuscular Diseases, Fondazione IRCCS, Istituto Nazionale Neurologico Carlo Besta, Milan, Italy.
Sabrina Sacconi, Université Côte d'Azur, Peripheral Nervous System and Muscle Department, Pasteur 2 Hospital, Centre Hospitalier Universitaire de Nice, Nice, France.
Kimiaki Utsugisawa, Department of Neurology, Hanamaki General Hospital, Hanamaki, Japan.
Tuan Vu, Department of Neurology, University of South Florida Morsani College of Medicine, Tampa, FL, USA.
Marion Boehnlein, UCB, Monheim, Germany.
Maryam Gayfieva, UCB, Slough, UK.
Bernhard Greve, UCB, Monheim, Germany.
Franz Woltering, UCB, Monheim, Germany.
John Vissing, Copenhagen Neuromuscular Center, Department of Neurology, Rigshospitalet, University of Copenhagen, Copenhagen, Denmark.

Document Type

Journal Article

Publication Date

3-19-2025

Journal

Journal of neurology

Volume

272

Issue

4

DOI

10.1007/s00415-025-12958-9

Keywords

FcRn inhibitor; Myasthenia gravis; Phase 3 clinical trial; Rozanolixizumab

Abstract

BACKGROUND: In the Phase 3 MycarinG study (NCT03971422), six once-weekly subcutaneous infusions of rozanolixizumab significantly improved myasthenia gravis (MG)-specific outcomes versus placebo in patients with acetylcholine receptor or muscle-specific tyrosine kinase autoantibody-positive generalized MG (gMG). Following completion of MycarinG, patients could enroll in the open-label extension MG0004 study (NCT04124965) to receive chronic weekly rozanolixizumab. METHODS: Patients were re-randomized 1:1 to once-weekly rozanolixizumab 7 or 10 mg/kg for up to 52 infusions. The primary endpoints were the occurrence of treatment-emergent adverse events (TEAEs) and TEAEs leading to rozanolixizumab discontinuation. After ≥6 visits/infusions patients could switch to the MG0007 study (NCT04650854) to receive cyclic rozanolixizumab treatment. RESULTS: In MG0004, 70 patients received rozanolixizumab 7 mg/kg (n = 35) or 10 mg/kg (n = 35). Mean treatment duration was 22.9 and 23.7 weeks, respectively, due to rollover into MG0007. TEAEs were reported in 60/70 (85.7%) patients; most were mild/moderate. The most frequently reported TEAEs were headache (25/70 [35.7%]), diarrhea (13/70 [18.6%]) and decreased blood immunoglobulin G (11/70 [15.7%]). There were no opportunistic, serious or severe infections, serious or severe hypersensitivity or injection-site reactions, any anaphylactic reactions or albumin or lipid abnormalities. Maximum mean reduction from baseline in MG Activities of Daily Living score was 3.1 in the 7 mg/kg group and 4.1 in the 10 mg/kg group. CONCLUSION: Chronic weekly rozanolixizumab for up to 52 infusions was generally well tolerated, and clinically relevant improvements across MG-specific outcomes were maintained, supporting the long-term use of rozanolixizumab in patients with gMG. TRIAL REGISTRATION: NCT04124965 (registered October 11, 2019).

APA Citation

Bril, Vera; Drużdż, Artur; Grosskreutz, Julian; Habib, Ali A.; Kaminski, Henry J.; Mantegazza, Renato; Sacconi, Sabrina; Utsugisawa, Kimiaki; Vu, Tuan; Boehnlein, Marion; Gayfieva, Maryam; Greve, Bernhard; Woltering, Franz; and Vissing, John, "Safety and efficacy of chronic weekly rozanolixizumab in generalized myasthenia gravis: the randomized open-label extension MG0004 study" (2025). GW Authored Works. Paper 6794.
https://hsrc.himmelfarb.gwu.edu/gwhpubs/6794

Department

Neurology