EU/3/14/1376: Orphan designation for the treatment of Niemann-Pick's disease, type C

arimoclomol citrate


On 19 November 2014, orphan designation (EU/3/14/1376) was granted by the European Commission to Orphazyme ApS, Denmark, for arimoclomol citrate for the treatment of Niemann-Pick disease, type C.

In October 2020, Orphazyme ApS changed name to Orphazyme A/S.

Key facts

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

Zevra Denmark A/S
Ole Maaloees Vej 3
2200 Copenhagen N

Review of designation

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

Update history

March 2023The sponsor’s name was changed from KemPharm Denmark A/S to Zevra Denmark A/S.
February 2023The sponsorship was transferred from Orphazyme A/S, Denmark to KemPharm Denmark A/S., Denmark.

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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