Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in January 2019 at the end of the 10-year period of market exclusivity.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

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

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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