Synonyms: RA-101495 | RA101495 | Zilbrysq®
zilucoplan is an approved drug (FDA & EMA (2023))
Compound class:
Peptide
Comment: Zilucoplan (RA101495) is a synthetic, macrocyclic peptide that blocks the cleavage of complement C5 into C5a and C5b which occurs as a consequence of activation of the classical, alternative, or lectin pathways. This mechanism of action inhibits formation of the membrane attack complex (MAC). It was developed by RaPharma for the treatment of tissue-based complement-mediated disorders.
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No information available. |
Summary of Clinical Use ![]() |
Zilucoplan was progressed to clinical evaluation in paroxysmal nocturnal hemoglobinuria (PNH) and generalized myasthenia gravis (gMG) [1]. Click here to link to ClinicalTrials.gov's full list of zilucoplan trials. The earliest approval for gMG was issued in Japan in September 2023 [4], with FDA approval following in October, and EMA authorisation in December. In response to the SARS-CoV-2 pandemic plans were made to evaluate zilucoplan in COVID-19 patients, as part of the UK's Accelerating COVID-19 Research and Development (ACCORD) initiative (June 2020). ACCORD aims to fast-track potential treatments for COVID-19 through early-stage clinical trials [5-6]. The first candidate agents included in the ACCORD study were 10478, MEDI3506 (tozorakimab, and anti-IL33 mAb), acalabrutinib, zilucoplan and nebulised heparin. Through this study researchers would aim to determine if the anti-inflammatory action mediated by zilucoplan has efficacy to reduce mortality in patients with severe COVID-19. In October 2023 the FDA approved zilucoplan to treat gMG in patients with acetylcholine receptor autoantibodies. |
Clinical Trials | |||||
Clinical Trial ID | Title | Type | Source | Comment | References |
NCT04225871 | Open-Label Extension of Zilucoplan in Subjects With Generalized Myasthenia Gravis | Phase 3 Interventional | UCB Pharma | ||
NCT04115293 | Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis | Phase 3 Interventional | UCB Pharma | 2 |
External links ![]() |
For extended ADME data see the following: European Medicines Agency (EMA) |