This medicine is now known as caplacizumab.
On 30 April 2009, orphan designation (EU/3/09/629) was granted by the European Commission to Ablynx NV, Belgium, for nanobody directed towards the human A1 domain of von Willebrand factor for the treatment of thrombotic thrombocytopenic purpura.
Nanobody directed towards the human A1 domain of von Willebrand factor has been authorised in the EU as Cablivi since 31 August 2018.
Nanobody directed towards the human A1 domain of von Willebrand factor (caplacizumab)
Treatment of thrombotic thrombocytopenic purpura
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Review of designation
The Committee for Orphan Medicinal Products reviewed the orphan designation of Cablivi at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.
More information is available in the orphan medicine assessment report .
Documents related to this orphan designation evaluation
EU/3/09/629: Public summary of positive opinion for orphan designation of nanobody directed towards the human A1 domain of von Willebrand factor for the treatment of thrombotic thrombocytopenic purpura (PDF/124.43 KB)
First published: 12/05/2009
Last updated: 06/04/2011
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: