EU/3/11/895: Orphan designation for the treatment of Niemann-Pick's disease, type C

hydroxy-propyl-beta-cyclodextrin (hydroxypropylbetadex)


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.

The sponsor's address was updated in September 2021.

Key facts

Active substance
hydroxy-propyl-beta-cyclodextrin (hydroxypropylbetadex)
Intented use
Treatment of Niemann-Pick's disease, type C
Orphan designation status
EU designation number
Date of designation

Boyd Consultants Limited
6th Floor
South Bank House
Barrow Street
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.

This medicine is now known as hydroxypropylbetadex.

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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