Caplacizumab is a von Willebrand factor (vWF)-directed antibody fragment used to treat acquired thrombotic thrombocytopenic purpura (aTTP).

Brand Names
Generic Name
DrugBank Accession Number

Caplacizumab, firstly called ALX-0081, is a humanized single-variable-domain immunoglobulin consisting of two identical humanized building blocks genetically linked by a three-alanine linker. Caplacizumab was developed by Ablynx, a Sanofi company and FDA approved on February 6, 2019,6 and approved previously by the EU in October 2018 as a combination therapy with plasma exchange and immunosuppression.1

Approved, Investigational
Biologic Classification
Protein Based Therapies
Monoclonal antibody (mAb)
Protein Structure
Protein Chemical Formula
Protein Average Weight
27880.0 Da
  1. Cablivi (caplacizumab) EMA label [File]
Download FASTA Format
  • Caplacizumab
  • caplacizumab-yhdp
External IDs
  • ALX 0081
  • ALX 0681
  • ALX-0081



Capacizumab is approved for the treatment of adults experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP) in conjunction with plasma exchange and immunosuppression in patients 18 years or older.1,6

aTTP is a rare autoimmune condition presented by a disruption of blood clotting order which is translated into systemic microvascular thrombosis leading to profound thrombocytopenia, hemolytic anemia and organ ischemia. It is caused by the production of autoantibodies against ADAMTS-13 which is the protein in charge of cleaving the von-Wilebrand factor. The lack of this process produces the generation of ultra large von Wilebrand multimers that bind to platelets and form microthrombi and causing thromboembolic complications.3

Previously, capacizumab was under review for the prevention of thrombosis in high-risk patients with acute coronary syndrome undergoing percutaneous coronary intervention but this indication was withdrawn.1

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Associated Conditions
Indication TypeIndicationCombined Product DetailsApproval LevelAge GroupPatient CharacteristicsDose Form
Used as adjunct in combination to treatAcquired thrombotic thrombocytopenic purpura••••••••••••••••••••••••••
Treatment ofAcquired thrombotic thrombocytopenic purpura (attp)••••••••••••••••••••••• •••••• •••••••••••••••••••••••• •••••••••• •••••• ••••••••• •••••• • •• •••••••••••• ••••••• ••• ••••••••
Contraindications & Blackbox Warnings
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In vitro studies have shown a caplacizumab-driven complete inhibition of platelet aggregation and in phase II clinical trials, it was shown to reduce the activity of the von Willebrand factor by 20% from treatment day 1 until treatment day 30. The level of von Willebrand factor in the plasma was also significantly reduced due to the clearance of the von Willebrand-caplacizumab complex.1

In phase III clinical trials, more than 50% of the tested individuals reached a platelet normal count. In these trials, it was observed as well a significant reduction in the incidence of aTTP1 as well as a significant reduction in the median time to response of about 39%.4 However, as caplacizumab does not target autoimmune response, relapses were observed after treatment discontinuation.2

The last clinical trial prior approval showed production of a platelet count of more than 150,000 per mcl after the cessation of plasma exchange therapy for 5 days as well as a reduction of patient recurrent thrombotic thrombocytopenic purpura and of disease-related death during treatment.6

Mechanism of action

Caplacizumab acts by targetting the A1 domain of the ultra-large von Willebrand factor which in order inhibits the interaction with the glycoprotein Ib-IX-V receptor in the platelets. Caplacizumab binds to von Willebrand factor with an affinity of 8.5 nM, thus it is very target specific.[5305] The blockage of the von Willebrand factor prevents the interaction between the von Willebrand factor and the platelets, hence, preventing platelet aggregation.1

Uvon Willebrand factorNot AvailableHumans

After intravenous administration of caplacizumab, the pharmacokinetic profile is non-linear and to follow a non-compartmental model as the pharmacokinetic profile of this drug is dependent on the expression of von Willebrand factor. After administration, caplacizumab is rapidly absorbed with a dose-dependent behavior. The peak concentration was reached after 6-7 hours1 and it presents a very high bioavailability reaching approximately 90%.10

The subcutaneous administration of a dose of 10 mg of caplacizumab produced a peak concentration of 528 ng/ml and an AUC of 7951 ng.h/ml.8

Volume of distribution

The reported volume of distribution of caplacizumab is 6.33 L.1

Protein binding

This antibody acts directly on plasma proteins and thus, this parameter is not significant for drug description.


Caplacizumab is degraded in the reticuloendothelial system to small peptides and amino acids which can be used for de-novo protein synthesis.5

Route of elimination

The elimination of caplacizumab is divided between target-driven disposition which is driven by the binding to the von Willebrand factor and non-target disposition driven by the combination of catabolism and renal elimination.10


The reported half-life is reported to be in the range of 16-27 hours.10


As the elimination is highly divided among hepatic, target-driven and renal elimination, the calculation of the clearance rate is not significant for drug description.

Adverse Effects
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Cases of overdose are represented by an increased risk of bleeding and in these cases, external administration of von Willebrand factor concentrate should be done.Label

To this point, there have not been performed studies regarding the effect on fertility, genotoxicity, or carcinogenicity

Not Available
Pharmacogenomic Effects/ADRs
Not Available


Drug Interactions
This information should not be interpreted without the help of a healthcare provider. If you believe you are experiencing an interaction, contact a healthcare provider immediately. The absence of an interaction does not necessarily mean no interactions exist.
AbciximabThe risk or severity of bleeding can be increased when Caplacizumab is combined with Abciximab.
AbrocitinibThe risk or severity of bleeding and thrombocytopenia can be increased when Caplacizumab is combined with Abrocitinib.
AceclofenacThe risk or severity of bleeding can be increased when Aceclofenac is combined with Caplacizumab.
AcemetacinThe risk or severity of bleeding can be increased when Acemetacin is combined with Caplacizumab.
AcenocoumarolThe risk or severity of bleeding can be increased when Caplacizumab is combined with Acenocoumarol.
Food Interactions
No interactions found.


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Brand Name Prescription Products
NameDosageStrengthRouteLabellerMarketing StartMarketing EndRegionImage
CabliviInjection, powder, for solution10 mgIntravenous; SubcutaneousAblynx Nv2021-01-12Not applicableEU flag
CabliviInjection, powder, for solution10 mgIntravenous; SubcutaneousAblynx Nv2021-01-12Not applicableEU flag
CabliviKit; Powder, for solution11 mg / vialIntravenous; SubcutaneousSanofi Aventis2020-09-04Not applicableCanada flag
CabliviInjection, powder, for solution10 mgIntravenous; SubcutaneousAblynx Nv2021-01-12Not applicableEU flag
CabliviInjection, powder, lyophilized, for solution; Kit11 mg/1mLIntravenous; SubcutaneousGenzyme Corporation2019-02-06Not applicableUS flag


ATC Codes
B01AX07 — Caplacizumab
Drug Categories
Chemical TaxonomyProvided by Classyfire
Not Available
Organic Compounds
Super Class
Organic Acids
Carboxylic Acids and Derivatives
Sub Class
Amino Acids, Peptides, and Analogues
Direct Parent
Alternative Parents
Not Available
Not Available
Molecular Framework
Not Available
External Descriptors
Not Available
Affected organisms
  • Humans and other mammals

Chemical Identifiers

CAS number


General References
  1. Duggan S: Caplacizumab: First Global Approval. Drugs. 2018 Oct;78(15):1639-1642. doi: 10.1007/s40265-018-0989-0. [Article]
  2. Coppo P, Cuker A, George JN: Thrombotic thrombocytopenic purpura: Toward targeted therapy and precision medicine. Res Pract Thromb Haemost. 2018 Nov 16;3(1):26-37. doi: 10.1002/rth2.12160. eCollection 2019 Jan. [Article]
  3. Peyvandi F, Scully M, Kremer Hovinga JA, Knobl P, Cataland S, De Beuf K, Callewaert F, De Winter H, Zeldin RK: Caplacizumab reduces the frequency of major thromboembolic events, exacerbations and death in patients with acquired thrombotic thrombocytopenic purpura. J Thromb Haemost. 2017 Jul;15(7):1448-1452. doi: 10.1111/jth.13716. Epub 2017 Jun 5. [Article]
  4. Peyvandi F, Scully M, Kremer Hovinga JA, Cataland S, Knobl P, Wu H, Artoni A, Westwood JP, Mansouri Taleghani M, Jilma B, Callewaert F, Ulrichts H, Duby C, Tersago D: Caplacizumab for Acquired Thrombotic Thrombocytopenic Purpura. N Engl J Med. 2016 Feb 11;374(6):511-22. doi: 10.1056/NEJMoa1505533. [Article]
  5. Tabrizi MA, Tseng CM, Roskos LK: Elimination mechanisms of therapeutic monoclonal antibodies. Drug Discov Today. 2006 Jan;11(1-2):81-8. doi: 10.1016/S1359-6446(05)03638-X. [Article]
  6. FDA news [Link]
  7. Guide to Pharmacology [Link]
  8. Clinical trials [Link]
  9. FDA Approved Drug Products: CABLIVI (caplacizumab-yhdp) injection [Link]
  10. Cablivi (caplacizumab) EMA label [File]
FDA label
Download (1.21 MB)
Download (92 KB)

Clinical Trials

Clinical Trials


Not Available
Not Available
Dosage Forms
Injection, powder, for solutionIntravenous; Subcutaneous10 mg
Injection, powder, for solutionParenteral; Subcutaneous10 MG
Injection, powder, lyophilized, for solution; kitIntravenous; Subcutaneous11 mg/1mL
Kit; powder, for solutionIntravenous; Subcutaneous11 mg / vial
Injection, powder, lyophilized, for solutionIntravenous; Subcutaneous10 mg
Not Available
Not Available


Experimental Properties
melting point (°C)61 ºC (Fab fragment)Vermeer, A.W.P. & Norde, W., Biophys. J. 78:394-404 (2000)
boiling point (°C)Fab fragment denaturates at 60 ºCArnoldus W. et al. (2000). Biophysical Journal. Vol 78. 394-404
water solubilitySolubleAcqua E. Can Caplacizumab be a potential first drug therapy for Thrombotic Thrombocytopenic Purpura?.
isoelectric point 6.6 - 7.2 Jin, et al. Electrophoresis. Sep;23(19):3385-91. (2002).


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Pharmacological action
General Function
Protein n-terminus binding
Specific Function
Important in the maintenance of hemostasis, it promotes adhesion of platelets to the sites of vascular injury by forming a molecular bridge between sub-endothelial collagen matrix and platelet-surf...
Gene Name
Uniprot ID
Uniprot Name
von Willebrand factor
Molecular Weight
309261.83 Da

Drug created at November 18, 2007 18:29 / Updated at June 03, 2022 07:24