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Crizanlizumab: First Approval

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Abstract

Crizanlizumab (Adakveo®; crizanlizumab-tmca) is an intravenously administered monoclonal antibody developed by Novartis Pharmaceuticals for the prevention of vaso-occlusive crises (VOCs) in patients with sickle cell disease. Crizanlizumab binds to P-selectin, thereby blocking its interaction with P-selectin glycoprotein ligand-1. In November 2019, crizanlizumab received its first global approval in the USA, where it is indicated to reduce the frequency of VOCs in adults and paediatric patients aged ≥ 16 years with sickle cell disease. The drug is also under regulatory review in the EU for the prevention of VOCs in patients with sickle cell disease. The use of crizanlizumab (in combination with ruxolitinib) in myelofibrosis is also being evaluated in Australia, Spain, Germany and Hungary. This article summarizes the milestones in the development of crizanlizumab leading to this first approval for the reduction of VOCs in patients with sickle cell disease.

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References

  1. Gardner RV. Sickle cell disease: advances in treatment. Ochsner J. 2018;18(4):377–89.

    Article  Google Scholar 

  2. Manwani D, Frenette PS. Vaso-occlusion in sickle cell disease: pathophysiology and novel targeted therapies. Blood. 2013;122(24):3892–8.

    Article  CAS  Google Scholar 

  3. Gutsaeva DR, Parkerson JB, Yerigenahally SD, et al. Inhibition of cell adhesion by anti-P-selectin aptamer: a new potential therapeutic agent for sickle cell disease. Blood. 2011;117(2):727–35.

    Article  CAS  Google Scholar 

  4. Shah N, Bhor M, Xie L, et al. Sickle cell disease complications: prevalence and resource utilization. PLoS One. 2019;14(7):e0214355.

    Article  CAS  Google Scholar 

  5. Selexys Pharmaceuticals. Selexys Pharmaceuticals initiates enrollment in phase I clinical study of SelG1 [media release]. 4 May 2011. http://www.selexys.com.

  6. US Food and Drug Administration. Crizanlizumab orphan drug designation. 2008. http://www.accessdata.fda.gov. Accessed 19 Dec 2019.

  7. Novartis. Novartis investigational therapy crizanlizumab (SEG101) receives FDA breakthrough therapy designation for the prevention of vaso-occlusive crises in sickle cell disease [media release]. 8 Jan 2019. http://www.novartis.com.

  8. Novartis. FDA accepts file and accelerates review of Novartis sickle cell disease medicine crizanlizumab (SEG101) [media release]. 16 Jul 2019. http://www.novartis.com.

  9. US Food and Drug Administration. FDA approves first targeted therapy to treat patients with painful complication of sickle cell disease [media release]. 15 Nov 2019. http://www.fda.gov.

  10. Novartis. ADAKVEO® (crizanlizumab-tmca) injection, for intravenous use: US prescribing information. 2019. http://www.accessdata.fda.gov. Accessed 19 Dec 2019.

  11. Novartis. Novartis delivers strong sales, double digit core operating income growth and launches Zolgensma and Piqray in second quarter; sales and profit guidance increased [media release]. 18 Jul 2019. http://www.novartis.com.

  12. Selexys Pharmaceuticals. Selexys Pharmaceuticals completes $23 M equity financing and signs agreement with global pharmaceutical company [media release]. 19 Sep 2012. http://www.selexys.com.

  13. Cytovance Biologics Inc. Cytovance® Biologics, Inc. announces manufacturing agreement with Selexys Pharmaceuticals, Corp. for phase II production of SelG1 [media release]. 13 Nov 2012. http://www.cytovance.com.

  14. Novartis. Novartis acquires Selexys Pharmaceuticals Corporation and SelG1 antibody for reduction of pain crises in sickle cell disease (SCD) [media release]. 21 Nov 2016. http://www.novartis.com.

  15. Selexys Pharmaceuticals. Selexys Pharmaceuticals completes phase I clinical study for sickle cell drug [media release]. 19 Dec 2011. http://www.selexys.com.

  16. Ataga KI, Kutlar A, Kanter J, et al. Crizanlizumab for the prevention of pain crises in sickle cell disease. N Engl J Med. 2017;376(5):429–39.

    Article  CAS  Google Scholar 

  17. Bailey M, Thompson M, Brown S. The impact of crizanlizumab on VOC-related medical facility visits [abstract no. PF715]. HemaSphere. 2019;3(Suppl 1):312–3.

    Article  Google Scholar 

  18. Kutlar A, Kanter J, Liles DK, et al. Effect of crizanlizumab on pain crises in subgroups of patients with sickle cell disease: a SUSTAIN study analysis. Am J Hematol. 2019;94(1):55–61.

    Article  CAS  Google Scholar 

  19. Liles D, Shah N, Scullin B, et al. SUCCESSOR: a multicenter retrospective noninterventional follow-up study in patients with sickle cell pain crises who previously participated in the SUSTAIN trial in the United States [abstract no. S853]. HemaSphere. 2019;3:380–1.

    Article  Google Scholar 

  20. Ataga KI, Kutlar A, Cancado R, et al. Crizanlizumab treatment is not associated with the development of proteinuria and hematuria in patients with sickle cell disease: a safety analysis from the SUSTAIN study [abstract no. PF712]. HemaSphere. 2018;2(Suppl 1):305–6.

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Correspondence to Hannah A. Blair.

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The preparation of this review was not supported by any external funding.

Conflict of interest

During the peer review process the manufacturer of the agent under review was offered an opportunity to comment on the article. Changes resulting from any comments received were made by the authors on the basis of scientific completeness and accuracy. Hannah A. Blair is a salaried employee of Adis International Ltd/Springer Nature, is responsible for the article content and declares no relevant conflicts of interest.

Additional information

Enhanced material for this AdisInsight Report can be found at https://doi.org/10.6084/m9.figshare.11401062.

This profile has been extracted and modified from the AdisInsight database. AdisInsight tracks drug development worldwide through the entire development process, from discovery, through pre-clinical and clinical studies to market launch and beyond.

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Blair, H.A. Crizanlizumab: First Approval. Drugs 80, 79–84 (2020). https://doi.org/10.1007/s40265-019-01254-2

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