The investigational drug zilucoplan is currently under review by the US Food and Drug Administration for the treatment of generalized myasthenia gravis (MG) following the submission of a New Drug Application, according to a recent press release.

Notably, the C5 inhibitor zilucoplan had already received orphan drug designation for MG in 2019. Furthermore, it has also recently received validation of its Marketing Authorization Application by the European Medicines Agency.

The data supporting its effectiveness and safety comes from the multicenter, phase 3, randomized, double-blind, placebo-controlled trial referred to as RAISE. The study has a current enrollment of 174 patients randomized in a 1:1 ratio to receive daily doses of 0.3 mg/kg of either placebo or zilucoplan for 12 weeks.

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The investigators set out to assess the therapeutic effect of the investigational therapy. Nonetheless, the MG-ADL Score is the primary endpoint of the study. In doing so, they utilized standardized clinical assessment tools such as the Myasthenia Gravis-Activities of Daily Living (MG-ADL), the Quantitative Myasthenia Gravis (QMG) score, and the Myasthenia Gravis Composite score.

Results from the RAISE trial revealed that patients receiving zilucoplan experienced statistically significant improvement compared to patients receiving a placebo. Patients in the therapeutic arm of the study presented with a 2 points increase in the MG-ADL.

Regarding safety, the study revealed that patients in the therapeutic arm had a similar incidence of treatment-emergent adverse events. The most common adverse effects included headache, diarrhea, and injection site bruising.

“If approved, zilucoplan has the potential to address the unmet need for people with [generalized myasthenia gravis (gMG)] by providing targeted improvements in signs and symptoms of gMG disease activity and severity. A benefit of targeted treatment is that it may help reduce the adverse events associated with nonspecific immunosuppressive treatment of gMG,” said Iris Loew-Friedrich, executive vice president and chief medical officer at UCB, the developer of the investigational therapy.

Developers expect a response in the last quarter of 2023, and regulatory filing in the rest of the world is expected in 2022.


UCB announces U.S. FDA acceptance of new drug application and EMA MAA validation for zilucoplan for the treatment of generalized myasthenia gravis in adult patients. News release. UCB; November 14, 2022.