EU/3/10/777: Orphan designation for the treatment of molybdenum-cofactor deficiency type A

Cyclic pyranopterin monophosphate (fosdenopterin)


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.

Key facts

Active substance
Cyclic pyranopterin monophosphate (fosdenopterin)
Intended use
Treatment of molybdenum-cofactor deficiency type A
Orphan designation status
EU designation number
Date of designation

TMC Pharma (EU) Limited
7a Durands Court
45 Parnell Street
X91 P381
Co. Waterford

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Nulibry at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.

More information is available in the PDF icon orphan medicine assessment report .

Update history

June 2023The sponsorship was transferred from Zydus France to TMC Pharma (EU) Limited, Ireland in June 2023.
November 2022The sponsorship was transferred from Comharsa Life Sciences Limited, Ireland to Zydus France, France.
September 2022The medicinal product was authorised in the EU as Nulibry.
May 2021The sponsorship was transferred to Comharsa Life Sciences Limited.
January 2019The sponsorship was transferred to Voisin Consulting S.A.R.L., France.
March 2013The sponsorship was transferred to Alexion Europe SAS, France.
January 2013Orphatec Pharmaceuticals GmbH changed name to Colbourne Pharmaceuticals GmbH.

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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