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Delgado J, Voltz C, Stain M, et al. The European Medicines Agency Review of Crizanlizumab for the Prevention of Recurrent Vaso-Occlusive Crises in Patients With Sickle Cell Disease. Hemasphere. 2021;5(7):e604doi: 10.1097/HS9.0000000000000604.
Delgado, J., Voltz, C., Stain, M., Lapveteläinen, T., Urach, S., Lähteenvuo, J., Penttilä, K., Gisselbrecht, C., Enzmann, H., & Pignatti, F. (2021). The European Medicines Agency Review of Crizanlizumab for the Prevention of Recurrent Vaso-Occlusive Crises in Patients With Sickle Cell Disease. HemaSphere, 5(7), e604. https://doi.org/10.1097/HS9.0000000000000604
Delgado, Julio, et al. "The European Medicines Agency Review of Crizanlizumab for the Prevention of Recurrent Vaso-Occlusive Crises in Patients With Sickle Cell Disease." HemaSphere vol. 5,7 (2021): e604. doi: https://doi.org/10.1097/HS9.0000000000000604
Delgado J, Voltz C, Stain M, Lapveteläinen T, Urach S, Lähteenvuo J, Penttilä K, Gisselbrecht C, Enzmann H, Pignatti F. The European Medicines Agency Review of Crizanlizumab for the Prevention of Recurrent Vaso-Occlusive Crises in Patients With Sickle Cell Disease. Hemasphere. 2021 Jun 28;5(7):e604. doi: 10.1097/HS9.0000000000000604. eCollection 2021 Jul. PMID: 34235401; PMCID: PMC8240778.
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Hemasphere. 2021 Jun 28;5(7):e604. doi: 10.1097/HS9.0000000000000604. eCollection 2021 Jul.

The European Medicines Agency Review of Crizanlizumab for the Prevention of Recurrent Vaso-Occlusive Crises in Patients With Sickle Cell Disease.

HemaSphere

Julio Delgado, Caroline Voltz, Milena Stain, Tuomo Lapveteläinen, Susanne Urach, Johanna Lähteenvuo, Karri Penttilä, Christian Gisselbrecht, Harald Enzmann, Francesco Pignatti

Affiliations

  1. Oncology & Haematology Office, European Medicines Agency, Amsterdam, The Netherlands.
  2. Department of Haematology, Hospital Clinic, Barcelona, Spain.
  3. Bundesamt fur Sicherheit im Gesundheitswesen, Vienna, Austria.
  4. Committe for Medicinal Products for Human Use, European Medicines Agency, Amsterdam, The Netherlands.
  5. Lääkealan turvallisuus- ja kehittämiskeskus, Fimea, Finland.
  6. Hopital Saint Louis, Paris, France.
  7. Bundesinstitut für Arzneimittel und Medizinprodukte, Bonn, Germany.

PMID: 34235401 PMCID: PMC8240778 DOI: 10.1097/HS9.0000000000000604

Abstract

Crizanlizumab is a monoclonal antibody that binds to P-selectin. On October 28, 2020, a conditional marketing authorization valid through the European Union (EU) was issued for crizanlizumab for the prevention of recurrent vaso-occlusive crises (VOCs) in patients with sickle cell disease aged 16 years or older. Crizanlizumab was evaluated in a phase 2, double-blind, placebo-controlled randomized multicenter trial comparing high-dose (5 mg/kg) crizanlizumab, low-dose (2.5 mg/kg) crizanlizumab and placebo in patients with a history of 2-10 VOCs in the previous year. Patients who were receiving concomitant hydroxycarbamide (HC) as well as those not receiving HC were included in the study. The primary endpoint of the trial was the annual rate of sickle cell-related pain crises as adjudicated by a central review committee. High-dose crizanlizumab led to a 45.3% lower median annual rate of sickle cell-related pain crises compared to placebo (

Copyright © 2021 the Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the European Hematology Association.

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