Caplacizumab

Overview

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, and approved previously by the EU in October 2018 as a combination therapy with plasma exchange and immunosuppression.

Indication

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. 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. 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high-risk patients with acute coronary syndrome undergoing percutaneous coronary intervention but this indication was withdrawn.

Associated Conditions

Acquired Thrombotic Thrombocytopenic Purpura (aTTP)

Clinical Trials

Title	Posted	Study ID	Phase	Status	Sponsor
Impact of Acute ITTP Therapies on Long Term Neurologic and Cognitive Outcomes in ITTP Survivors	2024/04/10	NCT06358703	N/A	Not yet recruiting	US Thrombotic Microangiopathy Alliance
Platelet Response to Caplacizumab in the Treatment of Acquired Thrombotic Thrombocytopenic Purpura	2023/05/25	NCT05876221	N/A	Recruiting	University of Cologne
A Retrospective, Observational Study on the Response to Caplacizumab Treatment in aTTP Patients: the Italian Experience (ROSCAPLI)	2023/03/27	NCT05785468	N/A	Recruiting	Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Caplacizumab and Immunosuppressive Therapy Without Firstline Therapeutic Plasma Exchange in Adults With Immune-mediated Thrombotic Thrombocytopenic Purpura	2022/07/21	NCT05468320	Phase 3	Completed	Sanofi
Retrospective Study on Caplacizumab-treated Pediatric Patients With Immune-mediated Thrombocytopenic Purpura (iTTP)	2022/03/02	NCT05263193	N/A	Completed	Sanofi
Retrospective Analysis of the Efficiency of Caplacizumab in the Treatment of aTTP	2021/08/02	NCT04985318	N/A	Recruiting	University of Cologne
Efficacy of a Personalized Caplacizumab Regimen Based on ADAMTS13 Activity Monitoring in Adult aTTP	2021/01/22	NCT04720261	Phase 2	UNKNOWN	University Hospital, Rouen
A Trial of Caplacizumab in Japanese Patients With Acquired Thrombotic Thrombocytopenic Purpura (aTTP)	2019/08/29	NCT04074187	Phase 2	Completed	Sanofi
Comparative Study of ALX-0081 Versus GPIIb/IIIa Inhibitor in High Risk Percutaneous Coronary Intervention (PCI) Patients	2009/11/24	NCT01020383	Phase 2	Completed	Ablynx, a Sanofi company

FDA Drug Approvals

Approved Product	Manufacturer	NDC Code	Route	Strength	Effective Date
No FDA approvals found for this drug.

EMA Drug Approvals

Approved Product	Authorization Holder	Status	Issued Date
Cablivi	Ablynx NV,Technologiepark 21,9052 Zwijnaarde,Belgium	Authorised	8/30/2018

HSA Drug Approvals

Approved Product	Manufacturer	Approval Number	Dosage Form	Strength	Approval Date
No HSA approvals found for this drug.

NMPA Drug Approvals

Approved Product	Company	Approval Number	Drug Type	Dosage Form	Approval Date
No NMPA approvals found for this drug.

PPB Drug Approvals

Approved Product	Registration No.	Company	Licence No.	Strength	Registration Date
No PPB approvals found for this drug.

TGA Drug Approvals

Approved Product	ARTG ID	Sponsor	Registration Type	Status	Registration Date
No TGA approvals found for this drug.

Health Canada Drug Approvals

Approved Product	Company	DIN	Dosage Form	Strength	Market Date
CABLIVI	sanofi-aventis canada inc	02496194	Powder For Solution , Kit - Subcutaneous , Intravenous	11 MG / VIAL	9/4/2020

CIMA AEMPS Drug Approvals

Approved Product	Company	Registration Number	Pharmaceutical Form	Prescription Type	Status
CABLIVI 10 MG POLVO Y DISOLVENTE PARA SOLUCION INYECTABLE	Ablynx	1181305001	POLVO Y DISOLVENTE PARA SOLUCIÓN INYECTABLE	Uso Hospitalario	Commercialized

Philippines FDA Drug Approvals

Approved Product	Company	License Number	Dosage Form	Strength	Approval Date
No Philippines FDA approvals found for this drug.

Saudi SFDA Drug Approvals

Approved Product	Company	License Number	Dosage Form	Strength	Approval Date
No Saudi SFDA approvals found for this drug.

Malaysia NPRA Drug Approvals

Approved Product	Company	Registration Number	Dosage Form	Strength	Approval Date
No Malaysia NPRA approvals found for this drug.

UK EMC Drug Information

Medicine Name	MA Holder	MA Number	Pharmaceutical Form	Active Ingredient	Authorization Date
No UK EMC drug information found for this drug.

Related News

EU Approves Sanofi's Cablivi as First Targeted Therapy for Rare Blood Clotting Disorder aTTP

7 years ago

The European Commission has authorized Cablivi (caplacizumab) as the first specific treatment for acquired thrombotic thrombocytopenic purpura (aTTP), a rare and life-threatening blood clotting disorder.

Help Us Improve

Your feedback helps us provide better drug information and insights.

AI-Powered Research

Premium Access