Switching to subcutaneous zilucoplan from intravenous complement component 5 inhibitors in generalised myasthenia gravis: a phase IIIb, open-label study

• Published in: Therapeutic advances in neurological disorders Vol. 18; p. 17562864251347283
• Main Authors: Freimer, Miriam, Desai, Urvi, Govindarajan, Raghav, Kang, Min K., Khan, Shaida, Khatri, Bhupendra, Levine, Todd, Macwan, Samir, Shieh, Perry B., Weiss, Michael D., Bloemers, Jos, Boroojerdi, Babak, Delicha, Eumorphia Maria, Lavrov, Andreea, Singh, Puneet, Howard, James F.
• Format: Journal Article
• Language: English
• Published: England SAGE Publications 01.01.2025; SAGE Publishing
• Subjects: Original Research; myasthenia gravis; complement inhibitor; eculizumab; ravulizumab; zilucoplan
• ISSN: 1756-2864; 1756-2856; 1756-2864
• DOI: 10.1177/17562864251347283

Experiences of patients with myasthenia gravis who switched their treatments from intravenous infusions of ravulizumab or eculizumab to self-administered daily injections of zilucoplan: effects on symptoms, safety, and patient preference Myasthenia gravis (MG) is a chronic autoimmune disease. Complement, part of the immune system, is overactive in MG. This damages the connections between muscles and nerves, causing muscle weakness. Symptoms can be reduced by blocking complement activity. Ravulizumab and eculizumab are complement inhibitors that are given as intravenous (into a vein) infusions by a healthcare professional in a hospital or clinic. Zilucoplan is another type of complement inhibitor, which patients can inject subcutaneously (under the skin) themselves. This study followed patients with MG who wanted to switch from ravulizumab or eculizumab to zilucoplan. Side effects were recorded throughout the study. Patients’ symptoms were measured to see if there was any change since switching treatment. All 26 patients who enrolled in the study had stable symptoms with their current treatment (which was ravulizumab in 10 patients, and eculizumab in 16 patients). Once enrolled, patients switched to daily zilucoplan for 12 weeks. Their main reasons for wanting to switch treatment were challenges with intravenous infusions, including travelling to hospital and long infusion times. Some patients also felt their current treatment was wearing off. After 12 weeks of zilucoplan treatment, symptoms across the group as a whole had improved. Symptoms had either improved or stayed the same in approximately 75% of patients. Symptom improvements were greatest in patients who switched from ravulizumab to zilucoplan. At the end of the study, 76.9% (20 out of 26) patients said they preferred injections with zilucoplan to intravenous infusions of their previous treatment. Side effects occurred in 73.1% of patients, which were mostly mild. Although the study was short and limited to a small number of patients, the results suggest that switching to zilucoplan is an option for patients with MG who would prefer self-injections to intravenous infusions. Longer-term studies are needed to confirm these findings.