Caplacizumab

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Caplacizumab
Monoclonal antibody
TypeSingle domain antibody
SourceHumanized
Targetvon Willebrand factor (VWF)
Names
Trade namesCablivi
Other namesALX-0081, caplacizumab-yhdp
Clinical data
Main usesThrombotic thrombocytopenic purpura (TTP)[1]
Side effectsNosebleeding, headaches, bleeding gums[2]
Pregnancy
category
  • AU: B1
  • US: N (Not classified yet)
Routes of
use
Intravenous, subcutaneous
External links
AHFS/Drugs.comMonograph
MedlinePlusa619030
Legal
License data
Legal status
  • AU: S4 (Prescription only)
  • UK: POM (Prescription only) [3]
  • US: ℞-only
  • EU: Rx-only
Chemical and physical data
FormulaC1213H1891N357O380S10
Molar mass27876.19 g·mol−1

Caplacizumab, sold under the brand name Cablivi, is a medication used to treat thrombotic thrombocytopenic purpura (TTP).[1] It is used with plasma exchange and immunosuppressive therapy.[1] It is given by injection into a vein or under the skin.[2]

Common side effects include nosebleeding, headaches, and bleeding gums.[2] Use in pregnancy may increase the risk of bleeding in the baby.[1] It is a small antibody that attaches to and inactivates von Willebrand factor.[2]

Caplacizumab was approved for medical use in Europe in 2018 and the United States in 2019.[2][1] In the United Kingdom a month of treatment costs about £125,000 as of 2021.[4] This amount in the United States costs about 230,000 USD.[5]

Medical uses

Dosage

It is given at a dose of 10 mg into a vein followed by 10 mg under the skin for at least 30 days.[2]

Mechanism of action

It is an anti-von Willebrand factor humanized immunoglobulin.[6] It acts by blocking platelet aggregation to reduce organ injury due to ischemia.[6]

Chemistry

It is a bivalent single-domain antibody (VHH).

History

The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.[7]

References

  1. 1.0 1.1 1.2 1.3 1.4 "Caplacizumab-yhdp Monograph for Professionals". Drugs.com. Retrieved 29 December 2021.
  2. 2.0 2.1 2.2 2.3 2.4 2.5 "Cablivi EPAR". European Medicines Agency (EMA). Archived from the original on 7 June 2020. Retrieved 7 September 2020.
  3. "Cablivi 10 mg powder and solvent for solution for injection - Summary of Product Characteristics (SmPC)". (emc). 22 June 2020. Archived from the original on 12 August 2021. Retrieved 22 August 2020.
  4. BNF 81: March-September 2021. BMJ Group and the Pharmaceutical Press. 2021. p. 1081. ISBN 978-0857114105.
  5. "Cablivi Prices, Coupons & Patient Assistance Programs". Drugs.com. Retrieved 29 December 2021.
  6. 6.0 6.1 "Immune Drug Tackles Microvascular Thrombosis Disorder. February 2016". Archived from the original on 16 January 2021. Retrieved 3 July 2021.
  7. "New Drug Therapy Approvals 2019". U.S. Food and Drug Administration. 31 December 2019. Archived from the original on 16 September 2020. Retrieved 15 September 2020.

External links

External sites:
Identifiers: