nach oben

Drugs

Erschienen in:

01.01.2019 | AdisInsight Report

Emapalumab: First Global Approval

verfasst von: Zaina T. Al-Salama

Erschienen in: Drugs | Ausgabe 1/2019

Einloggen, um Zugang zu erhalten

Abstract

Emapalumab-Izsg (hereafter referred to as emapalumab) [Gamifant^®] is a monoclonal antibody directed against interferon gamma that is available as an intravenous infusion. Emapalumab is being developed by Novimmune and Swedish Orphan Biovitrum for the treatment of haemophagocytic lymphohistiocytosis (HLH). In November 2018, emapalumab received its first global approval in the USA, for the treatment of paediatric (newborn and older) and adult patients with primary HLH, who have refractory, recurrent or progressive disease or intolerance to conventional HLH therapy. Emapalumab is under regulatory review in the EU for the treatment of primary HLH. This article summarizes the milestones in the development of emapalumab leading to this first global approval for HLH in the USA.

Daver N, McClain K, Allen CE, et al. A consensus review on malignancy-associated hemophagocytic lymphohistiocytosis in adults. Cancer. 2017;123(17):3229–40.CrossRef

UK National Institute for Health Research. Emapalumab for primary haemophagocytic lymphohistiocytosis. 2018. http://www.io.nihr.ac.uk/. Accessed 7 Dec 2018.

De Min C, Buatois V, Chatel L, et al. Interferon gamma (IFNg) is the driving mediator of secondary hemophagocytic lymphohistiocytosis (sHLH) in TLR9-mediated pathogenesis in mice and is correlated to disease parameters in children [abstract no. 3076]. Arthritis Rheumatol. 2015;67(Suppl. 10):3691–2.

Prencipe G, Caiello I, Pascarella A, et al. Neutralization of IFN-gamma reverts clinical and laboratory features in a mouse model of macrophage activation syndrome. J Allergy Clin Immunol. 2018;141(4):1439–49.CrossRef

Jordan MB, Hildeman D, Kappler J, et al. An animal model of hemophagocytic lymphohistiocytosis (HLH): CD8+ T cells and interferon gamma are essential for the disorder. Blood. 2004;104(3):735–43.CrossRef

Pachlopnik Schmid J, Ho CH, Chrétien F, et al. Neutralization of IFNgamma defeats haemophagocytosis in LCMV-infected perforin- and Rab27a-deficient mice. EMBO Mol Med. 2009;1(2):112–24.CrossRef

Novimmune SA. GAMIFANT^TM (emapalumab-lzsg): prescribing information. 2018. https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/761107lbl.pdf. Accessed 7 Dec 2018.

US Food and Drug Administration (FDA). FDA approves first treatment specifically for patients with rare and life-threatening type of immune disease [media release]. 2018. https://www.fda.gov/NewsEvents/. Accessed 7 Dec 2018.

Swedish Orphan Biovitrum. Sobi^TM completes acquisition of the global rights for emapalumab from Novimmune [media release]. 2018. http://www.sobi.com. Accessed 7 Dec 2018.

10.

Swedish Orphan Biovitrum. Sobi^TM strengthens inflammation franchise by acquiring global rights for emapalumab from Novimmune [media release]. 2018. http://www.sobi.com. Accessed 7 Dec 2018.

11.

Jordan M, Locatelli F, Allen C, et al. A novel targeted approach to the treatment of hemophagocytic lymphohistiocytosis (HLH) with an anti-interferon gamma (IFNgamma) monoclonal antibody (MAB), NI-0501: first results from a pilot phase 2 study in children with primary HLH [abstract plus presentation]. Blood. 2015;126(23):LBA-3.

12.

Hatterer E, Richard F, Malinge P, et al. Investigating the novel mechanism of action for NI-0501, a human interferon gamma monoclonal antibody [abstract no. P156]. Cytokine. 2012;59(3):570.CrossRef

13.

Locatelli F, Jordan M, Allen C, et al. Anti-interferon gamma monoclonal antibody NI-0501: a new option to render allogeneic HSCT for HLH safer and more effective? [abstract no. O147]. Bone Marrow Transplant. 2016;51(Suppl):S86.

14.

Jordan MB, Locatelli F, Allen C, et al. A novel targeted approach to the treatment of hemophagocytic lymphohistiocytosis (HLH) with NI-0501, an anti-interferon gamma monoclonal antibody [abstract no. 4511]. J Clin Immunol. 2016;36(3):282.

15.

Locatelli F, Jordan MB, Allen CE, et al. Safety and efficacy of emapalumab in pediatric patients with primary hemophagocytic lymphohistiocytosis [abstract no. LBA-6]. In: The American Society of Hematology Annual Meeting. 2018.

16.

De Min C, Jacqmin P, Laveille C, et al. Innovative approach for the identification of an appropriate dose regimen of a targeted treatment, NI-0501, an anti-interferon gamma (IFNg) antibody, in patients with hemophagocytic lymphohistiocytosis (HLH) [abstract no. 3097]. Arthritis Rheumatol. 2015;67(Suppl. 10):3712.

17.

Locatelli F, Jordan M, Allen C, et al. Interferon gamma (IFN-gamma) neutralization as a valuable therapeutic target in primary HLH [abstract no. P343]. Haematologica. 2016;101(Suppl. 1):114–5.

Titel: Emapalumab: First Global Approval
verfasst von: Zaina T. Al-Salama
Publikationsdatum: 01.01.2019
Verlag: Springer International Publishing
Erschienen in: Drugs / Ausgabe 1/2019
Print ISSN: 0012-6667
Elektronische ISSN: 1179-1950
DOI: https://doi.org/10.1007/s40265-018-1046-8

Springer Medizin

Abstract

Bitte loggen Sie sich ein, um Zugang zu diesem Inhalt zu erhalten

Weitere Artikel der Ausgabe 1/2019

Recent Developments in Male Contraception

Targeting Angiogenesis in Colorectal Carcinoma

Lorlatinib: First Global Approval

Immune Checkpoint Inhibitors in the Treatment of Gastroesophageal Cancer

Targeted Systemic Treatment of Neuroendocrine Tumors: Current Options and Future Perspectives

Disease-Modifying Therapies in Type 1 Diabetes: A Look into the Future of Diabetes Practice