On 16 February 2006, orphan designation (EU/3/06/351) was granted by the European Commission to Actelion Registration Ltd, United Kingdom, for miglustat for the treatment of Niemann-Pick disease, type C.
Miglustat for the treatment of Niemann-Pick disease, type C has been authorised in the EU as Zavesca since 26 January 2009.
The sponsorship was transferred to Janssen-Cilag International N.V., Belgium, in September 2018.
Treatment of Niemann-Pick's disease, type C
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Review of designation
Documents related to this orphan designation evaluation
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: