On 20 September 2010, orphan designation (EU/3/10/777) was granted by the European Commission to Orphatec Pharmaceuticals GmbH, Germany, for cyclic pyranopterin monophosphate for the treatment of molybdenum-cofactor deficiency type A.
This medicine is now known as fosdenopterin.
The medicinal product has been authorised in the EU as Nulibry since 15 September 2022.
Cyclic pyranopterin monophosphate (fosdenopterin)
Treatment of molybdenum-cofactor deficiency type A
|Orphan designation status||
|EU designation number||
|Date of designation||
Review of designation
|June 2023||The sponsorship was transferred from Zydus France to TMC Pharma (EU) Limited, Ireland in June 2023.|
|November 2022||The sponsorship was transferred from Comharsa Life Sciences Limited, Ireland to Zydus France, France.|
|September 2022||The medicinal product was authorised in the EU as Nulibry.|
|May 2021||The sponsorship was transferred to Comharsa Life Sciences Limited.|
|January 2019||The sponsorship was transferred to Voisin Consulting S.A.R.L., France.|
|March 2013||The sponsorship was transferred to Alexion Europe SAS, France.|
|January 2013||Orphatec Pharmaceuticals GmbH changed name to Colbourne Pharmaceuticals GmbH.|
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: