On 11 January 2012, orphan designation (EU/3/11/945) was granted by the European Commission to Dr Ulrich Granzer, Germany, for ornithine phenylacetate for the treatment of acute liver failure.

The sponsorship was transferred to Voisin Consulting S.A.R.L., France, in September 2014, to Amiculum Consulting Limited, United Kingdom, in February 2017, and to Mallinckrodt Pharmaceuticals Ireland Limited, Ireland, in April 2018.

Key facts

Active substance
Ornithine phenylacetate
Disease / condition
Treatment of acute liver failure
Date of decision
Orphan decision number

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

Mallinckrodt Pharmaceuticals Ireland Limited
College Business & Technology Park
Cruiserath, Blanchardstown
Dublin 15, Ireland
Tel. +41 41 726 3045

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

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