EU/3/13/1124: Orphan designation for the treatment of Niemann-Pick's disease, type C



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.

The sponsorship was transferred to Rapport Global Strategic Services Ireland Limited in December 2021.

Key facts

Active substance
Intended use
Treatment of Niemann-Pick's disease, type C
Orphan designation status
EU designation number
Date of designation

Rapport Global Strategic Services Ireland Limited
22 Northumberland Road
Dublin 4

Review of designation

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

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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