Caplacizumab Explained

Type:mab
Mab Type:sdAb
Source:zu
Target:von Willebrand factor (VWF)
Tradename:Cablivi
Dailymedid:Caplacizumab
Pregnancy Au:B1
Routes Of Administration:Intravenous, subcutaneous
Atc Prefix:B01
Atc Suffix:AX07
Legal Au:S4
Legal Ca:Rx-only
Legal Ca Comment:/ Schedule D[1] [2]
Legal Uk:POM
Legal Uk Comment:[3]
Legal Us:Rx-only
Legal Eu:Rx-only
Cas Number:915810-67-2
Drugbank:DB06081
Chemspiderid:none
Unii:2R27AB6766
Kegg:D11160
Synonyms:ALX-0081, caplacizumab-yhdp
C:1213
H:1891
N:357
O:380
S:10

Caplacizumab (INN; trade name Cablivi) is a bivalent single-domain antibody (VHH) designed for the treatment of thrombotic thrombocytopenic purpura (TTP) and thrombosis.[4] [5] [6] [7]

This drug was developed by Ablynx NV. On 30 August 2018, it was approved in the European Union for the "treatment of adults experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), in conjunction with plasma exchange and immunosuppression".[8]

It is an anti-von Willebrand factor humanized immunoglobulin.[9] It acts by blocking platelet aggregation to reduce organ injury due to ischemia.[9] Results of the phase II TITAN trial have been reported.[9]

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

In February 2019, caplacizumab-yhdp (Cablivi, Ablynx NV) was approved in the United States for the treatment of adults with acquired thrombotic thrombocytopenic purpura (aTTP). The drug is used in combination with plasma exchange and immunosuppressive therapy.[10] [11] The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.[12]

While it has been adopted for upfront use in TTP treatment at a number of institutions, its cost-effectiveness has been questioned.[13] Use of caplacizumab without plasmapheresis has been reported in select patients.[14] The MAYARI study was designed to evaluate the effectiveness of this option.[15]

Notes and References

  1. Web site: Cablivi Product information . Health Canada . 25 April 2012 . 29 May 2022.
  2. Web site: Summary Basis of Decision (SBD) for Cablivi . Health Canada . 23 October 2014 . 29 May 2022.
  3. Web site: Cablivi 10 mg powder and solvent for solution for injection - Summary of Product Characteristics (SmPC) . (emc) . 22 June 2020 . 22 August 2020.
  4. http://www.ama-assn.org/resources/doc/usan/x-pub/caplacizumab.pdf Statement On A Nonproprietary Name Adopted By The USAN Council - Caplacizumab
  5. World Health Organization (WHO) . International Nonproprietary Names for Pharmaceutical Substances (INN). Proposed INN: List 106 . WHO Drug Information . 25 . 4 . 2011 .
  6. Gómez-Seguí I, Fernández-Zarzoso M, de la Rubia J . A critical evaluation of caplacizumab for the treatment of acquired thrombotic thrombocytopenic purpura . Expert Review of Hematology . 13 . 11 . 1153–1164 . November 2020 . 32876503 . 10.1080/17474086.2020.1819230 . 221468324 .
  7. Poullin P, Bornet C, Veyradier A, Coppo P . Caplacizumab to treat immune-mediated thrombotic thrombocytopenic purpura . Drugs of Today . Barcelona, Spain . 55 . 6 . 367–376 . June 2019 . 31250841 . 10.1358/dot.2019.55.6.2989843 . 195761938 .
  8. Web site: Cablivi EPAR . European Medicines Agency (EMA) . 7 September 2020.
  9. http://www.medpagetoday.com/Cardiology/VenousThrombosis/56154 Immune Drug Tackles Microvascular Thrombosis Disorder. February 2016
  10. FDA approves first therapy for the treatment of adult patients with a rare blood clotting disorder . U.S. Food and Drug Administration (FDA) . 6 February 2019 . https://web.archive.org/web/20191123082824/https://www.fda.gov/news-events/press-announcements/fda-approves-first-therapy-treatment-adult-patients-rare-blood-clotting-disorder . 23 November 2019 . live . 22 November 2019.
  11. Web site: Drug Trials Snapshots: Cablivi . U.S. Food and Drug Administration (FDA) . 11 February 2019 . https://web.archive.org/web/20191123082855/https://www.fda.gov/drugs/drug-trials-snapshots-cablivi . 23 November 2019 . live . 22 November 2019.
  12. Web site: New Drug Therapy Approvals 2019 . U.S. Food and Drug Administration . 31 December 2019 . 15 September 2020.
  13. Goshua G, Sinha P, Hendrickson JE, Tormey C, Bendapudi PK, Lee AI. Cost effectiveness of caplacizumab in acquired thrombotic thrombocytopenic purpura. Blood. 2021 Feb 18;137(7):969-976. doi: 10.1182/blood.2020006052. PMID: 33280030; PMCID: PMC7918179.
  14. Völker LA, Brinkkoetter PT, Knöbl PN, Krstic M, Kaufeld J, Menne J, Buxhofer-Ausch V, Miesbach W. Treatment of acquired thrombotic thrombocytopenic purpura without plasma exchange in selected patients under caplacizumab. J Thromb Haemost. 2020 Nov;18(11):3061-3066. doi: 10.1111/jth.15045. Epub 2020 Sep 6. PMID: 32757435; PMCID: PMC7692904.
  15. Web site: NCT05468320 . Caplacizumab and Immunosuppressive Therapy Without Firstline Therapeutic Plasma Exchange in Adults With Immune-mediated Thrombotic Thrombocytopenic Purpura (MAYARI) . Clinicaltrials.gov . NCI . August 4, 2024.

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