caplacizumab   Click here for help

GtoPdb Ligand ID: 9123

Synonyms: ALX-0081 | anti-vWF Nanobody | Cablivi® | caplacizumab-yhdp
Approved drug
caplacizumab is an approved drug (EMA (2018), FDA (2019))
Compound class: Antibody
Comment: Caplacizumab (ALX-0081) is a nanobody targeting von Willebrand factor (vWF) [5], with anti-thrombotic action [1]. NanobodyTM technology was commercially developed by Ablynx. Nanobodies are antibody derivatives comprising only the single variable domains (variable heavy, VH) whilst retaining full antigen binding capacity. Their relatively small size (compared to complete antibodies) provides the potential for alternative modes of delivery and the possibility of brain penetrance.
Annotated peptide sequences for this therapeutuc are available from its IMGT/mAb-DB record. BLAST protein sequence analysis of the peptide sequence submitted with the INN document for caplacizumab reveals a 100% match with SEQ ID NO: 90 claimed in patent US7807162 [4], which is preferred humanized variant 12A2H1 in the patent document.
No information available.
Summary of Clinical Use Click here for help
Caplacizumab was granted EMA marketing authorisation in 2018, as a therapeutic to treat the rare blood clotting disorder, acquired thrombotic thrombocytopenic purpura (aTTP).
Preclinical efficacy was reported in [5]. Phase 2 clinical outcomes in patients with aTTP (NCT01151423) were reported in [2] and Phase 3 results from NCT02553317 were published in early 2019 [3]. Caplacizumab is to be used in combination with plasma exchange and immunosuppressive therapy. FDA approval followed in Feb. 2019.
Mechanism Of Action and Pharmacodynamic Effects Click here for help
Caplacizumab binds to the A1 domain of vWF, and specifically inhibits interaction of vWF with the platelet GPIb receptor complex, the first step in platelet adhesion to the vessel wall.
Clinical Trials
Clinical Trial ID Title Type Source Comment References
NCT01151423 Study to Assess Efficacy and Safety of Anti-von Willebrand Factor (vWF) Nanobody in Patients With Acquired Thrombotic Thrombocytopenic Purpura (aTTP) Phase 2 Interventional Ablynx
NCT02553317 Phase III Trial With Caplacizumab in Patients With Acquired Thrombotic Thrombocytopenic Purpura Phase 3 Interventional Ablynx
External links Click here for help