Treatment-emergent adverse events (TEAEs) occurred in 22.6% of patients (7 of 31), and all were mild and nonserious. Injection-site pain was the most common TEAE, reported in 3 patients (9.7%). At day 14, the median score on the satisfaction with self-injection domain of the Self-Injection Assessment Questionnaire, which ranges from 0 to 10, was 8.9 (range, 6-10).1,2
UCB also cited a human factors validation study of 100 participants, including patients with gMG or ocular symptoms, caregivers, and health care professionals, in which 90% completed an injection with the autoinjector on their first attempt without training.1
Counseling and Dispensing Considerations
The device does not change zilucoplan’s safety profile. The drug carries a boxed warning for serious meningococcal infections, and patients must complete or update vaccination against serogroups A, C, W, Y, and B at least 2 weeks before the first dose unless the risks of delaying therapy outweigh the risk of infection. Patients remain at increased risk even if they develop antibodies after vaccination. Under the REMS program, pharmacies that dispense the drug must be certified and must verify that prescribers are certified, and patients must carry a Patient Safety Card during treatment and for 2 months after stopping it.3
Dosing is based on actual body weight: 16.6 mg once daily for patients weighing less than 56 kg, 23 mg for 56 kg to less than 77 kg, and 32.4 mg for 77 kg or more. Patients who miss a dose should inject it as soon as possible, then resume their regular schedule without taking more than 1 dose per day. Because pancreatitis and pancreatic cysts have been reported, baseline lipase and amylase levels are recommended before treatment starts, and patients should be counseled on their increased susceptibility to other encapsulated bacteria, including Streptococcus pneumoniae and Neisseria gonorrhoeae.3
A Second Self-Injection Option
Zilucoplan received initial US approval in 2023 based on a 12-week, randomized, placebo-controlled study in which it significantly improved Myasthenia Gravis Activities of Daily Living total scores versus placebo (least-squares mean difference, -2.09; 95% CI, -3.24 to -0.95; P < .001).3
gMG is a rare autoimmune neuromuscular disease with a global prevalence of 150 to 350 cases per 1 million people, and symptoms can include double vision, drooping eyelids, difficulty swallowing, chewing, and talking, and life-threatening weakness of the respiratory muscles.1
