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.
|Disease / condition||
Treatment of Niemann-Pick disease, type C
|Date of decision||
|Orphan decision number||
Review of designation
Sponsor's contact details
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;