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.
Treatment of Niemann-Pick's disease, type C
|Orphan designation status||
|EU designation number||
|Date of designation||
Review of designation
|March 2023||The sponsor’s name was changed from KemPharm Denmark A/S to Zevra Denmark A/S.|
|February 2023||The sponsorship was transferred from Orphazyme A/S, Denmark to KemPharm Denmark A/S., Denmark.|
Documents related to this orphan designation evaluation
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: