EU/3/14/1376

About

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
Disease / condition
Treatment of Niemann-Pick's disease, type C
Date of first decision
19/11/2014
Outcome
Positive
EU designation number
EU/3/14/1376

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

Orphazyme A/S
Ole Maaløes Vej 3
2200 Copenhagen N
Hovedstaden
Denmark
Tel. +45 39 17 82 72
E-mail: contact@orphazyme.com

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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