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Open Access 14.12.2023 | AdisInsight Report

Zilucoplan: First Approval

verfasst von: Matt Shirley

Erschienen in: Drugs | Ausgabe 1/2024

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Abstract

Zilucoplan (Zilbrysq^®) is a subcutaneously administered macrocyclic peptide inhibitor of complement component 5 (C5 inhibitor) being developed by UCB for the treatment of generalised myasthenia gravis (gMG). Zilucoplan received its first approval, in Japan, in September 2023 for the treatment of gMG in adult patients who inadequately respond to steroids or other immunosuppressants and are positive for anti-acetylcholine receptor (AChR) antibodies. Subsequently, zilucoplan was approved in the USA in October 2023 for the treatment of gMG in adult patients who are anti-AChR antibody positive and in the EU in December 2023 as an add-on to standard therapy for the treatment of gMG in adult patients who are anti-AChR antibody positive. Zilucoplan is also currently under regulatory review in Australia and Canada for use in the treatment of gMG. This article summarises the milestones in the development of zilucoplan leading to this first approval for gMG.

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US FDA. ZILBRYSQ (zilucoplan) injection, for subcutaneous use: US prescribing information. 2023. https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/216834s000lbl.pdf. Accessed 10 Nov 2023.

Pharmaceuticals and Medical Devices Agency. ZILBRYSQ^® syringe for SC injections: Japanese prescribing information. 2023. https://www.pmda.go.jp/english/review-services/reviews/approved-information/drugs/0003.html. Accessed 10 Nov 2023.

Howard JF Jr, Bresch S, Genge A, et al. Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study. Lancet Neurol. 2023;22(5):395–406.CrossRefPubMed

UCB. UCB announces approval of RYSTIGGO[^®] (rozanolixizumab) and ZILBRYSQ[^®] (zilucoplan) for the treatment of adult patients with generalized myasthenia gravis in Japan [media release]. 2023. https://www.ucb.com/stories-media/Press-Releases/article/UCB-announces-approval-of-RYSTIGGOR-rozanolixizumab-and-ZILBRYSQR-zilucoplan-for-the-treatment-of-adult-patients-with-generalized-myasthenia-gravis-in-Japan. Accessed 25 Sep 2023.

US FDA. Zilbrysq NDA approval. 2023. https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2023/216834Orig1s000ltr.pdf. Accessed 10 Nov 2023.

UCB. UCB announces European Commission approval of ZILBRYSQ® (zilucoplan) for the treatment of adults with generalized Myasthenia Gravis [media release]. https://www.ucb.com/sites/default/files/press_files/ac12afd8f326ccb1.pdf. Accessed 5 Dec 2023.

Kulasekararaj AG, Lehtinen A-E, Forsyth C, et al. Phase II trials of zilucoplan in paroxysmal nocturnal hemoglobinuria. Haematologica. 2023.

Mammen AL, Amato AA, Dimachkie MM, et al. Zilucoplan in immune-mediated necrotising myopathy: a phase 2, randomised, double-blind, placebo-controlled, multicentre trial. Lancet Rheumatol. 2023;5(2):e67-76.CrossRefPubMedPubMedCentral

Paganoni S, Berry J, Quintana M, et al. Results from the first four regimens of the HEALEY ALS Platform Trial [abstract]. Neurology. 2023;100(17 Suppl 2):PL5.004.

10.

De Leeuw E, Van Damme KFA, Declercq J, et al. Efficacy and safety of the investigational complement C5 inhibitor zilucoplan in patients hospitalized with COVID-19: an open-label randomized controlled trial. Respir Res. 2022;23(1):202.CrossRefPubMedPubMedCentral

11.

Wilkinson T, Dixon R, Page C, et al. ACCORD: a multicentre, seamless, phase 2 adaptive randomisation platform study to assess the efficacy and safety of multiple candidate agents for the treatment of COVID-19 in hospitalised patients: a structured summary of a study protocol for a randomised controlled trial. Trials. 2020;21(1):691.CrossRefPubMedPubMedCentral

12.

Ako A, Glatt S, Lalla M, et al. Efficacy and safety of zilucoplan for the treatment of COVID-19 in hospitalized patients: part of the COMMUNITY platform trial. Am J Respir Crit Care Med. 2023;207(1):A1648.

13.

UCB. UCB completes the acquisition of Ra Pharmaceuticals - to deliver differentiated therapies to patients [media release]. https://www.ucb.com/stories-media/Press-Releases/article/UCB-completes-the-acquisition-of-Ra-Pharmaceuticals-to-deliver-differentiated-therapies-to-patients. Accessed 2 Apr 2020.

14.

Ra Pharmaceuticals. Ra Pharmaceuticals reports third quarter 2018 financial results and provides corporate update [media release]. 2018. http://www.rapharma.com. Accessed 19 Nov 2018.

15.

Tang GQ, Tang Y, Dhamnaskar K, et al. Zilucoplan, a macrocyclic peptide inhibitor of human complement component 5, uses a dual mode of action to prevent terminal complement pathway activation. Front Immunol. 2023;14:1213920.CrossRefPubMedPubMedCentral

16.

Howard JF Jr, Nowak RJ, Wolfe GI, et al. Clinical effects of the self-administered subcutaneous complement inhibitor zilucoplan in patients with moderate to severe generalized myasthenia gravis: results of a phase 2 randomized, double-blind, placebo-controlled, multicenter clinical trial. JAMA Neurol. 2020;77(5):582–92.CrossRefPubMed

17.

Muppidi S, Wolfe GI, Conaway M, et al. MG-ADL: still a relevant outcome measure. Muscle Nerve. 2011;44(5):727–31.CrossRefPubMed

18.

Genge A, Hussain Y, Kaminski HJ, et al. Safety and tolerability of zilucoplan in RAISE-XT: a multicenter, open-label extension study in patients with myasthenia gravis [abstract plus poster]. Muscle Nerve. 2022;66(S1):S131.

19.

Leite MI, Bresch S, Freimer M, et al. Long-term safety, efficacy and self-injection satisfaction with zilucoplan in myasthenia gravis: an interim analysis of RAISE-XT [abstract and poster EPO-219]. In: EAN 2023. 2023.

Titel: Zilucoplan: First Approval
verfasst von: Matt Shirley
Publikationsdatum: 14.12.2023
Verlag: Springer International Publishing
Erschienen in: Drugs / Ausgabe 1/2024
Print ISSN: 0012-6667
Elektronische ISSN: 1179-1950
DOI: https://doi.org/10.1007/s40265-023-01977-3

Live-Webinar "Urologie und Sexualmedizin in der Praxis"

Springer Medizin

Zilucoplan: First Approval

Abstract

Live-Webinar "Urologie und Sexualmedizin in der Praxis"

Springer Medizin

Abstract

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