EU Approves Self-Administration Routes for Rozanolixizumab for Myasthenia Gravis

Donatello Crocetta

According to a recent announcement, UCB’s rozanolixizumab (Rystiggo), a medication for various forms of myasthenia gravis (MG), has gained EU approval for 2 new self-administration methods, including an infusion pump or manual push with a syringe. The belief is that the new self-administered subcutaneous treatment may offer several advantages, including high patient satisfaction, sense of control, and increased independence.¹

Rozanolixizumab is a humanized high-affinity, anti-human neonatal Fc receptor (FcRn) monoclonal antibody targeting immunoglobulin G. The newly approved self-administration methods were based on data from a phase 3, open-label, crossover study (NCT05681715) in which patients were randomly assigned to once-weekly rozanolixizumab for 18 consecutive weeks consisting of a 6-week self-administration training period, followed by two 6-week self-administration periods.

"For people living with gMG, unpredictable symptoms can have a significant impact on daily life, leading to patients feeling vulnerable and lacking control. Subcutaneous self-administration may help address these challenges, enhancing patient autonomy and satisfaction by reducing the need for frequent clinic visits,” Donatello Crocetta, chief medical officer and head of Global Medical Affairs at UCB, said in a statement.¹ "We welcome the EU approval for self-administration of rozanolixizumab in Europe, marking another significant step forward in our ongoing commitment to improving the lives of people living with gMG."

The crossover study, known as MG0020, tested whether patients can safely self-administer rozanolixizumab using a syringe driver and manual push method. Following the training period, 55 patients were randomly assigned 1:1 to the syringe driver or manual push self-administration method, subsequently crossing over to the alternative method. Led by Rachana K. Gandhi Mehta, MBBS, an assistant professor of neurology at the Wake Forest School of Medicine, the primary end point was successful self-administration of rozanolixizumab, evaluated by a healthcare professional at the end of each 6-week self-administration period.

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Each vial of rozanolixizumab solution for injection contains 140 mg/ml, with available vial sizes of 2 ml (280 mg), 3 ml (420 mg), 4 ml (560 mg), and 6 ml (840 mg). Across the MG studies, the most commonly reported adverse reactions to rozanolixizumab were headache (48.4%), diarrhea (25.0%), and pyrexia (12.5%). To date, rozanolixizumab remains contraindicated for patients with hypersensitivity to the active substance or to any of the excipients.

Rozanolixizumab was first approved by the FDA in June 2023 as a new treatment for both anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive gMG, the most common subtypes of the disease. The basis for the approval was data from the phase 3 MycarinG study (NCT03971422), in which rozanolixizumab-treated patients showed significant reductions in the primary end point of Myasthenia Gravis-Activities of Daily Living (MG-ADL) scores (P <.001) over a 43-day period.^2,3

In the MycarinG study, 200 patients were randomized into three groups to receive rozanolixizumab 7 mg/kg (n = 66), 10 mg/kg (n = 67), or placebo (n = 67) for 6 weeks, followed by an 8-week observation period. The treatment demonstrated significant effects on secondary endpoints, including the Quantitative Myasthenia Gravis (QMG) score, which measures muscle weakness. By day 43, the QMG total score showed statistically significant improvements in the rozanolixizumab groups, with point changes of –5.4 and –6.7 in the 7 mg/kg and 10 mg/kg groups, respectively, compared to –1.9 in the placebo group (P < .001).

During the 2024 American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) meeting, Mehta sat down with NeurologyLive® to discuss MG0200 and its unique build. In the conversation below, she provided clinical insight on how the study came to be and the advantages the syringe driver may bring. Additionally, she commented on how this study spoke to the progress in treating patients with gMG.

REFERENCES
1. RYSTIGGO[®]▼(rozanolixizumab), for generalized myasthenia gravis (gMG), receives EU approval for two new administration methods. News release. UCB. January 31, 2025. Accessed February 3, 2025. https://www.ucb.com/newsroom/press-releases/article/rystiggorvrozanolixizumab-for-generalized-myasthenia-gravis-gmg-receives-eu-approval-for-two-new-administration-methods
2. UCB announces US FDA approval of Rystiggo (rozanolixizumab-noli) for the treatment of adults with generalized myasthenia gravis. News release. June 27, 2023. Accessed February 3, 2025. https://www.prnewswire.com/news-releases/ucb-announces-us-fda-approval-of-rystiggo-rozanolixizumab-noli-for-the-treatment-of-adults-with-generalized-myasthenia-gravis-301864023.html
2. Bril V, Druzdz A, Grosskreutz J, et al. Safety and efficacy of rozanolixizumab in patients with generalized myasthenia gravis (MycarinG): a randomized, double-blind, placebo-controlled, adaptive phase 3 study. Lancet Neurol. 2023;22(5):383-394. doi:10.1016/S1474-4422(23)00077-7.