CHMP positive for zilucoplan as add on to standard therapy for generalized myasthenia gravis in adult patients who are anti acetylcholine receptor (AChR) antibody positive

The CHMP’s positive opinion is now being reviewed by the European Commission, which grants centralized marketing authorizations for medicinal products in the EU. Feedback from the commission is anticipated before the end of the year.

Following approval, zilucoplan will be the first once-daily subcutaneous (SC), targeted peptide inhibitor of complement component 5 (C5 inhibitor) and the only self-administered gMG therapy for use by adult patients with AChR antibody positive gMG. As a C5 inhibitor, zilucoplan inhibits complement-mediated damage to the neuromuscular junction through its targeted dual mechanism of action. Benefits of SC self-administration can include reduced traveling time to and from hospitals, decreased interference with work obligations, and increased independence. Unlike monoclonal antibody C5 inhibitors, as a peptide, zilucoplan can be used concomitantly with intravenous immunoglobulin and plasma exchange, without the need for supplemental dosing.

UCB’s RAISE study , published earlier this year in the Lancet Neurology journal, demonstrated that zilucoplan delivered rapid, consistent, statistically significant and clinically meaningful benefits in different patient-and-clinician-reported outcomes - Myasthenia Gravis-Activities of Daily Living (MG-ADL) score, Quantitative Myasthenia Gravis (QMG) score, Myasthenia Gravis Composite (MGC) score and Myasthenia Gravis Quality of Life 15-item scale (MG-QoL15r)* - at week 12 in a broad population of mild to severe adult patients with AChR antibody positive gMG. Additionally, rapid improvements in fatigue were observed as an exploratory endpoint.