Caplacizumab

Last updated

Caplacizumab
Monoclonal antibody
Type Single domain antibody
Source Humanized
Target von Willebrand factor (VWF)
Clinical data
Trade names Cablivi
Other namesALX-0081, caplacizumab-yhdp
AHFS/Drugs.com Monograph
MedlinePlus a619030
License data
Pregnancy
category
  • AU:B1
Routes of
administration 		Intravenous, subcutaneous
ATC code
Legal status
Legal status
Identifiers
CAS Number
DrugBank
ChemSpider
  • none
UNII
KEGG
Chemical and physical data
Formula C1213H1891N357O380S10
Molar mass 27876.19 g·mol−1

Caplacizumab, sold under the brand name Cablivi, is a monoclonal antibody used for the treatment of thrombotic thrombocytopenic purpura. [4] [6] [7] It is a von Willebrand factor-directed antibody fragment. [4] It is given via intravenous injection followed by subcutaneous injection. [4] Caplacizumab was developed by Ablynx NV.

Contents

Caplacizumab was authorized for medical use in the European Union in August 2018, [5] and approved for medical use in the United States in February 2019. [8] [9] The US Food and Drug Administration considers it to be a first-in-class medication. [10]

Medical uses

Caplacizumab is indicated for the treatment of acquired thrombotic thrombocytopenic purpura, in combination with plasma exchange and immunosuppressive therapy. [4] [5] [11]

Side effects

Common adverse effects include injection site reactions, reported in 3–6% of patients in the HERCULES and TITAN trials. [12]

Society and culture

Caplacizumab was authorized for medical use in the European Union in August 2018, [5] and approved for medical use in the United States in February 2019. [8] [11]

Economics

The cost-effectiveness has been questioned. [13]

Names

Caplacizumab is the international nonproprietary name [14] and the United States Adopted Name. [15]

Research

Use of caplacizumab without plasmapheresis has been reported. [16] The MAYARI study was designed to evaluate the effectiveness of this option. [17]

References

  1. ↑ "Cablivi Product information". Health Canada. 25 April 2012. Retrieved 29 May 2022.
  2. ↑ "Summary Basis of Decision (SBD) for Cablivi". Health Canada. 23 October 2014. Retrieved 29 May 2022.
  3. ↑ "Cablivi 10 mg powder and solvent for solution for injection - Summary of Product Characteristics (SmPC)". (emc). 22 June 2020. Retrieved 22 August 2020.
  4. 1 2 3 4 5 "Cablivi- caplacizumab kit". DailyMed. 4 November 2024. Retrieved 9 January 2026.
  5. 1 2 3 4 "Cablivi EPAR". European Medicines Agency (EMA). 10 September 2018. Retrieved 7 September 2020.
  6. ↑ Gómez-Seguí I, Fernández-Zarzoso M, de la Rubia J (November 2020). "A critical evaluation of caplacizumab for the treatment of acquired thrombotic thrombocytopenic purpura". Expert Review of Hematology. 13 (11): 1153–64. doi:10.1080/17474086.2020.1819230. PMID   32876503. S2CID   221468324.
  7. ↑ Poullin P, Bornet C, Veyradier A, Coppo P (June 2019). "Caplacizumab to treat immune-mediated thrombotic thrombocytopenic purpura". Drugs of Today. 55 (6). Barcelona, Spain: 367–376. doi:10.1358/dot.2019.55.6.2989843. PMID   31250841. S2CID   195761938.
  8. 1 2 "FDA approves first therapy for the treatment of adult patients with a rare blood clotting disorder". U.S. Food and Drug Administration (FDA) (Press release). 6 February 2019. Archived from the original on 23 November 2019. Retrieved 22 November 2019.PD-icon.svg This article incorporates text from this source, which is in the public domain .
  9. ↑ "Drug Trials Snapshots: Cablivi". U.S. Food and Drug Administration (FDA). 11 February 2019. Archived from the original on 23 November 2019. Retrieved 22 November 2019.PD-icon.svg This article incorporates text from this source, which is in the public domain .
  10. ↑ "New Drug Therapy Approvals 2019". U.S. Food and Drug Administration. 31 December 2019. Archived from the original on 2 May 2020. Retrieved 15 September 2020.
  11. 1 2 "FDA approves therapy for rare blood disorder in pediatric patients 12 years and older". U.S. Food and Drug Administration (FDA). 5 January 2026. Retrieved 9 January 2026.PD-icon.svg This article incorporates text from this source, which is in the public domain .
  12. ↑ FDA Professional Drug Information
  13. ↑ Goshua G, Sinha P, Hendrickson JE, Tormey C, Bendapudi PK, Lee AI (February 2021). "Cost effectiveness of caplacizumab in acquired thrombotic thrombocytopenic purpura". Blood. 137 (7): 969–976. doi:10.1182/blood.2020006052. PMC   7918179 . PMID   33280030.
  14. ↑ World Health Organization (WHO) (2011). "International Nonproprietary Names for Pharmaceutical Substances (INN). Proposed INN: List 106" (PDF). WHO Drug Information. 25 (4).
  15. ↑ "Statement On A Nonproprietary Name Adopted By The USAN Council - Caplacizumab" (PDF). American Medical Association.
  16. ↑ Völker LA, Brinkkoetter PT, Knöbl PN, Krstic M, Kaufeld J, Menne J, et al. (November 2020). "Treatment of acquired thrombotic thrombocytopenic purpura without plasma exchange in selected patients under caplacizumab". J Thromb Haemost. 18 (11): 3061–6. doi:10.1111/jth.15045. PMC   7692904 . PMID   32757435.
  17. ↑ Clinical trial number NCT05468320 for "Caplacizumab and Immunosuppressive Therapy Without Firstline Therapeutic Plasma Exchange in Adults With Immune-mediated Thrombotic Thrombocytopenic Purpura (MAYARI)" at ClinicalTrials.gov
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