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Caplacizumab

From Wikipedia, the free encyclopedia

Caplacizumab
Monoclonal antibody
TypeSingle domain antibody
SourceHumanized
Targetvon Willebrand factor (VWF)
Clinical data
Trade namesCablivi
Other namesALX-0081, caplacizumab-yhdp
AHFS/Drugs.comMonograph
MedlinePlusa619030
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
FormulaC1213H1891N357O380S10
Molar mass27876.19 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.

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

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

Side effects

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

Society and culture

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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

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The cost-effectiveness has been questioned.[13]

Names

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

Research

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

References

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  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. ^ a b c d e "Cablivi- caplacizumab kit". DailyMed. 4 November 2024. Retrieved 9 January 2026.
  5. ^ a b c d "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. ^ a b "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. Public Domain 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. Public Domain 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. ^ a b "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. Public Domain 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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  • Clinical trial number NCT02553317 for "Phase III Trial With Caplacizumab in Patients With Acquired Thrombotic Thrombocytopenic Purpura (HERCULES)" at ClinicalTrials.gov
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