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Drug

Caplacizumab

Approved · EMA
ClassVon Willebrand factor inhibitorEmerging researchLate-stage development

Also known as Cablivi.

RxNorm2110605ChEMBLCHEMBL2109624ATCB01AX07UNII2R27AB6766
1
Research papers
von Willebrand factor
Primary target
1
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

An Open-label, Single-arm, Multicenter Study to Evaluate the Efficacy and Safety of Caplacizumab and Immunosuppressive Therapy Without Firstline Therapeutic Plasma Exchange in Adults With Immune-mediated Thrombotic Thrombocytopenic Purpura

Clinical trial2025-12-30Results posted · ClinicalTrials.gov

Efficacy of a Personalized Caplacizumab Regimen Based on ADAMTS13 Activity Monitoring in Adult Acquired Thrombotic Thrombocytopenic Purpura: A Phase II, Multicenter Non-inferiority Single-arm Study.

Clinical trial2023-05-16Terminated · ClinicalTrials.gov

A Phase III Double-blind, Randomized, Parallel Group, Multicenter Placebo-controlled Trial to Study the Efficacy and Safety of Caplacizumab in Patients With Acquired Thrombotic Thrombocytopenic Purpura

Clinical trial2023-04-05Results posted · ClinicalTrials.gov

A Phase II, Single-blind, Randomized, Placebo-controlled Trial to Study the Efficacy and Safety of Anti-von Willebrand Factor Nanobody Administered as Adjunctive Treatment to Patients With Acquired Thrombotic Thrombocytopenic Purpura

Clinical trial2023-04-04Results posted · ClinicalTrials.gov

Prospective Follow-up Study for Patients Who Completed Study ALX0681-C301 (HERCULES) to Evaluate Long-term Safety and Efficacy of Caplacizumab (Post-HERCULES)

Clinical trial2022-03-28Results posted · ClinicalTrials.gov

An Open-label Multicenter Trial to Study the Efficacy and Safety of Caplacizumab in Japanese Patients With Acquired Thrombotic Thrombocytopenic Purpura

Clinical trial2021-05-19Completed · ClinicalTrials.gov

The Therapeutic Potential of Nanobodies.

Research2020-02-01BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy

Approval: Cablivi (EMA)

Regulatory2018-08-30EMA

A Phase I, Single Center, Open-Label, Randomized, Single Dose Cross-Over Study in Healthy Male Subjects to Investigate the Bioequivalence and Tolerability of Liquid and Reconstituted Lyophilized Subcutaneous Formulations of Caplacizumab.

Clinical trial2014-11-14Completed · ClinicalTrials.gov

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Caplacizumab
Aliases & brands
Cablivi
RxNorm CUI
2110605
ChEMBL ID
CHEMBL2109624
ATC codes
B01AX07
UNII
2R27AB6766
Primary mechanism
Von Willebrand factor inhibitor
Regulatory jurisdictions
ema

Pharmacology & targets

1 target

Known molecular targets and mechanisms supported by curated pharmacology databases.

von Willebrand factor
VWF · P04275
INHIBITORvon Willebrand factor inhibitor

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval
2018-08-30
Latest approval
2018-08-30
Authorities
EMA
Total events
1
emaEuropean Medicines Agency· 1 event
2018-08-30Approval
Approval: Cablivi (EMA)
Indication: Cablivi is indicated for the treatment of adults experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), in conjunction with plasma exchange and… Show full indication

Cablivi is indicated for the treatment of adults experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), in conjunction with plasma exchange and immunosuppression.

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

7 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
7
registered trials across all phases
LATEST COMPLETION 2024
4
Late-stage (III+)
6
Completed
1
Discontinued
PHASE DISTRIBUTIONn = 7
Phase 11Phase 22Phase 2 / 31Phase 33

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

An Open-label, Single-arm, Multicenter Study to Evaluate the Efficacy and Safety of Caplacizumab and Immunosuppressive Therapy Without Firstline Therapeutic Plasma Exchange in Adults With Immune-mediated Thrombotic Thrombocytopenic Purpura

CompletedPH IIISanofin=512024

An Open-label Multicenter Trial to Study the Efficacy and Safety of Caplacizumab in Japanese Patients With Acquired Thrombotic Thrombocytopenic Purpura

CompletedPH II/IIISanofin=212021

Prospective Follow-up Study for Patients Who Completed Study ALX0681-C301 (HERCULES) to Evaluate Long-term Safety and Efficacy of Caplacizumab (Post-HERCULES)

CompletedPH IIISanofin=1042020

A Phase III Double-blind, Randomized, Parallel Group, Multicenter Placebo-controlled Trial to Study the Efficacy and Safety of Caplacizumab in Patients With Acquired Thrombotic Thrombocytopenic Purpura

CompletedPH IIIAblynx, a Sanofi companyn=1452017

Recent completions

Trials that read out recently, adding to the completed evidence base.

An Open-label, Single-arm, Multicenter Study to Evaluate the Efficacy and Safety of Caplacizumab and Immunosuppressive Therapy Without Firstline Therapeutic Plasma Exchange in Adults With Immune-mediated Thrombotic Thrombocytopenic Purpura

CompletedPH IIISanofin=512024

An Open-label Multicenter Trial to Study the Efficacy and Safety of Caplacizumab in Japanese Patients With Acquired Thrombotic Thrombocytopenic Purpura

CompletedPH II/IIISanofin=212021

Prospective Follow-up Study for Patients Who Completed Study ALX0681-C301 (HERCULES) to Evaluate Long-term Safety and Efficacy of Caplacizumab (Post-HERCULES)

CompletedPH IIISanofin=1042020

A Phase III Double-blind, Randomized, Parallel Group, Multicenter Placebo-controlled Trial to Study the Efficacy and Safety of Caplacizumab in Patients With Acquired Thrombotic Thrombocytopenic Purpura

CompletedPH IIIAblynx, a Sanofi companyn=1452017

A Phase I, Single Center, Open-Label, Randomized, Single Dose Cross-Over Study in Healthy Male Subjects to Investigate the Bioequivalence and Tolerability of Liquid and Reconstituted Lyophilized Subcutaneous Formulations of Caplacizumab.

CompletedPH IAblynx, a Sanofi companyn=242014

A Phase II, Single-blind, Randomized, Placebo-controlled Trial to Study the Efficacy and Safety of Anti-von Willebrand Factor Nanobody Administered as Adjunctive Treatment to Patients With Acquired Thrombotic Thrombocytopenic Purpura

CompletedPH IIAblynx, a Sanofi companyn=752014

Research activity

1 papers

Key research shaping understanding of this drug, combining the latest publications with the most influential evidence.

Most influential

The Therapeutic Potential of Nanobodies.

BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2020 · 535 cites
Recent papers

The Therapeutic Potential of Nanobodies.

BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2020 · 535 cites
Journals, researchers & institutions
Top journals
  • BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy1
Leading researchers
  • Jovčevska I1
  • Muyldermans S1
Leading institutions
free-text, unnormalised
  • Medical Center for Molecular Biology1
  • Vrije Universiteit Brussel1
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
  • Europe PMC — research literature
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.