EU/3/06/351: Orphan designation for the treatment of Niemann-Pick's disease, type C



Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in January 2019 at the end of the 10-year period of market exclusivity.

On 16 February 2006, orphan designation (EU/3/06/351) was granted by the European Commission to Actelion Registration Ltd, United Kingdom, for miglustat for the treatment of Niemann-Pick disease, type C.

Miglustat for the treatment of Niemann-Pick disease, type C has been authorised in the EU as Zavesca since 26 January 2009.

The sponsorship was transferred to Janssen-Cilag International N.V., Belgium, in September 2018.

Key facts

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

Janssen-Cilag International N.V.
Turnhoutseweg, 30
2340 Beerse
Tel. +32 146 031 89

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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