EU/3/12/1034: Orphan designation for the treatment of sickle cell disease

Humanised monoclonal antibody against P-selectin (crizanlizumab)


This medicine is now known as crizanlizumab.

On 9 August 2012, orphan designation (EU/3/12/1034) was granted by the European Commission to Quintiles Ireland Ltd, Ireland, for humanised monoclonal antibody against P-selectin for the treatment of sickle-cell disease.

The sponsorship was transferred to Novartis Europharm Limited, United Kingdom, in March 2017 and subsequently to Novartis Europharm Limited, Ireland, in May 2018.

The medicinal product has been authorised in the EU as Adakveo since 28 October 2020.

Key facts

Active substance
Humanised monoclonal antibody against P-selectin (crizanlizumab)
Intended use
Treatment of sickle cell disease
Orphan designation status
EU designation number
Date of designation
Novartis Europharm Limited
Vista Building
Elm Park
Merrion Road
Dublin 4
Tel. +41 61 324 11 11 (Switzerland)

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Humanised monoclonal antibody against P-selectin at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.

More information is available in the PDF icon orphan medicine assessment report .

Documents related to this orphan designation evaluation

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

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:

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