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Caplacizumab
Monoclonal antibody
Type	Single domain antibody
Source	Humanized
Target	von Willebrand factor (VWF)
Clinical data
Trade names	Cablivi
Other names	ALX-0081, caplacizumab-yhdp
AHFS/Drugs.com	Monograph
MedlinePlus	a619030
License data	US DailyMed: Caplacizumab;
Pregnancy; category	AU: B1; ;
Routes of; administration	Intravenous, subcutaneous
ATC code	B01AX07 (WHO) ;
Legal status
Legal status	AU: S4 (Prescription only); CA: ℞-only / Schedule D; UK: POM (Prescription only); US: ℞-only; EU: Rx-only;
Identifiers
CAS Number	915810-67-2;
DrugBank	DB06081;
ChemSpider	none;
UNII	2R27AB6766;
KEGG	D11160;
Chemical and physical data
Formula	C1213H1891N357O380S10
Molar mass	27876.19 g·mol−1

Caplacizumab (INN; trade name Cablivi) is a bivalent single-domain antibody (VHH) designed for the treatment of thrombotic thrombocytopenic purpura (TTP) and thrombosis.^[5]^[6]^[7]^[8]

This drug was developed by Ablynx NV.^[9] 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".^[10]

It is an anti-von Willebrand factor humanized immunoglobulin.^[11] It acts by blocking platelet aggregation to reduce organ injury due to ischemia.^[11] Results of the phase II TITAN trial have been reported.^[11]

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

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.^[13]^[14] The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.^[15]

While it has been adopted for upfront use in TTP treatment at a number of institutions, its cost-effectiveness has been questioned.^[16] Use of caplacizumab without plasmapheresis has been reported in select patients.^[17] The MAYARI study was designed to evaluate the effectiveness of this option.^[18]

References

^ "Cablivi Product information". Health Canada. 25 April 2012. Retrieved 29 May 2022.
^ "Summary Basis of Decision (SBD) for Cablivi". Health Canada. 23 October 2014. Retrieved 29 May 2022.
^ "Cablivi 10 mg powder and solvent for solution for injection - Summary of Product Characteristics (SmPC)". (emc). 22 June 2020. Retrieved 22 August 2020.
^ "Cablivi EPAR". European Medicines Agency (EMA). 30 April 2009. Retrieved 3 December 2024.
^ Statement On A Nonproprietary Name Adopted By The USAN Council - Caplacizumab, American Medical Association.
^ World Health Organization (WHO) (2011). "International Nonproprietary Names for Pharmaceutical Substances (INN). Proposed INN: List 106" (PDF). WHO Drug Information. 25 (4).
^ 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): 1153–1164. doi:10.1080/17474086.2020.1819230. PMID 32876503. S2CID 221468324.
^ 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.
^ Clinical trial number NCT02553317 for "A Trial With Caplacizumab in Patients With Acquired Thrombotic Thrombocytopenic Purpura (HERCULES)" at ClinicalTrials.gov
^ "Cablivi EPAR". European Medicines Agency (EMA). Retrieved 7 September 2020.
^ ^a ^b ^c Immune Drug Tackles Microvascular Thrombosis Disorder. February 2016
^ FDA Professional Drug Information
^ "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. This article incorporates text from this source, which is in the public domain.
^ "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. This article incorporates text from this source, which is in the public domain.
^ "New Drug Therapy Approvals 2019". U.S. Food and Drug Administration. 31 December 2019. Retrieved 15 September 2020.
^ 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.
^ 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.
^ NCT05468320. "Caplacizumab and Immunosuppressive Therapy Without Firstline Therapeutic Plasma Exchange in Adults With Immune-mediated Thrombotic Thrombocytopenic Purpura (MAYARI)". Clinicaltrials.gov. NCI. Retrieved 4 August 2024.{{cite web}}: CS1 maint: numeric names: authors list (link)

[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] "Cablivi EPAR". European Medicines Agency (EMA). 30 April 2009. Retrieved 3 December 2024.

[5] Statement On A Nonproprietary Name Adopted By The USAN Council - Caplacizumab, American Medical Association.

[6] World Health Organization (WHO) (2011). "International Nonproprietary Names for Pharmaceutical Substances (INN). Proposed INN: List 106" (PDF). WHO Drug Information. 25 (4).

[pmid32876503-7] 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): 1153–1164. doi:10.1080/17474086.2020.1819230. PMID 32876503. S2CID 221468324.

[pmid31250841-8] 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.

[9] Clinical trial number NCT02553317 for "A Trial With Caplacizumab in Patients With Acquired Thrombotic Thrombocytopenic Purpura (HERCULES)" at ClinicalTrials.gov

[10] "Cablivi EPAR". European Medicines Agency (EMA). Retrieved 7 September 2020.

[Titan2016-11] Immune Drug Tackles Microvascular Thrombosis Disorder. February 2016

[12] FDA Professional Drug Information

[13] "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. This article incorporates text from this source, which is in the public domain.

[14] "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. This article incorporates text from this source, which is in the public domain.

[15] "New Drug Therapy Approvals 2019". U.S. Food and Drug Administration. 31 December 2019. Retrieved 15 September 2020.

[16] 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.

[17] 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.

[MAYARI-18] NCT05468320. "Caplacizumab and Immunosuppressive Therapy Without Firstline Therapeutic Plasma Exchange in Adults With Immune-mediated Thrombotic Thrombocytopenic Purpura (MAYARI)". Clinicaltrials.gov. NCI. Retrieved 4 August 2024.{{cite web}}: CS1 maint: numeric names: authors list (link)

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Caplacizumab

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