Objective: The purpose of this study is to describe the clinical experience including effectiveness and side effects of once-daily subcutaneous zilucoplan use in 10 patients with acetylcholine receptor-positive generalized myasthenia gravis (AChR +vegMG).
Background: Zilucoplan is a macrocytic peptide complement C5 inhibitor developed for use in AChR +vegMG. There is currently limited data characterizing the real-world use of zilucoplan since its approval in the US in October 2023.
Design/Methods: This is a retrospective chart review of 10 AChR +ve gMG patients who were treated with at least 8 weeks of once-daily subcutaneous zilucoplan and had both pre- and post- zilucoplan myasthenia gravis activities of daily living (MG-ADL) scores. Information regarding patient demographics, MG clinical history, and adverse events were reported. An improvement in MG-ADL of 2 or more was considered clinically meaningful.
Results: A total of 10 patients (M:3, F:7) with a mean age of 48.7 (± 39) were included in this cohort. Mean duration of symptoms since diagnosis 4.6 (±9) years. MGFA class of one 2A, four 2B, and five 3A were included. Median MG-ADL before starting zilucoplan was 9.5. Median MG-ADL at the end of 1 month was 7 and at the end of two months was 6. Side effects included urticaria at the injection site in one patient and headache in another patient. Both were self-limiting and did not lead to treatment discontinuation.
Conclusions: Zilucoplan led to clinically meaningful improvement in MG-ADL in all AChR +ve gMG patients in this cohort at the end of two months with mild side effects.