On 26 April 2013, orphan designation (EU/3/13/1124) was granted by the European Commission to International Niemann-Pick Disease Alliance (INPDA), the United Kingdom, for 2-hydroxypropyl-β-cyclodextrin for the treatment of Niemann-Pick disease, type C.
The sponsorship was transferred to Vtesse Europe Limited, United Kingdom, in February 2015.
The sponsorship was transferred to Mallinckrodt Pharmaceuticals Ireland Limited - Ireland, in September 2018.
For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.
|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;