On 30 August 2011, orphan designation (EU/3/11/895) was granted by the European Commission to Susan French, United Kingdom, for hydroxy-propyl-beta-cyclodextrin for the treatment of Niemann-Pick disease, type C.

The sponsorship was transferred to Medical Need Europe AB, Sweden, in March 2015.

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 Alan Boyd Consultants Ltd, United Kingdom, in September 2018.

The sponsorship was transferred to Boyd Consultants Limited, Ireland, in March 2019.

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

Boyd Consultants Limited
Dublin City University
DCU Invent
Dublin 9
Tel. +44 1270 270010

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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