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.

Key facts

Active substance
Disease / condition
Treatment of Niemann-Pick's disease, type C
Date of first decision
EU designation number

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor's contact details

Mallinckrodt Pharmaceuticals Ireland Limited
College Business & Technology Park
Cruiserath, Blanchardstown
Dublin 15, Ireland
Tel. +41 41 726 3045

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;

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