EU/3/20/2337 - orphan designation for treatment of limb-girdle muscular dystrophyDate of designation: 19 October 2020PositiveLast updated: 27 February 2023OrphanHuman
Changing the name or address of a sponsorLast updated: 20 January 2023HumanRegulatory and procedural guidanceRare diseasesResearch and development
EU/3/18/2057 - orphan designation for treatment of acute myeloid leukaemiaDate of designation: 24 August 2018WithdrawnLast updated: 22 November 2022OrphanHuman
EU/3/01/077 - orphan designation for treatment of short bowel syndromeDate of designation: 12 December 2001PositiveLast updated: 12 October 2022OrphanHuman
EU/3/17/1948 - orphan designation for treatment of familial cerebral cavernous malformationsDate of designation: 12 December 2017PositiveLast updated: 11 October 2022OrphanHuman
EU/3/20/2264 - orphan designation for treatment of autosomal recessive congenital ichthyosisDate of designation: 24 March 2020WithdrawnLast updated: 7 June 2022OrphanHuman
EU/3/16/1663 - orphan designation for treatment of interstitial cystitisDate of designation: 30 May 2016PositiveLast updated: 16 May 2022OrphanHuman
EU/3/07/478 - orphan designation for treatment of Hodgkin's lymphomaDate of designation: 14 September 2007WithdrawnLast updated: 10 May 2022OrphanHuman
Seventh industry stakeholder platform on research and development support Online European Medicines Agency, Amsterdam, the Netherlands Start date: 23 November 2021 End date: 23 November 2021 EventHumanInnovationRare diseasesPaediatricsResearch and developmentScientific advice
EU/3/15/1437 - orphan designation for treatment of acute myeloid leukaemiaDate of designation: 12 February 2015PositiveLast updated: 7 March 2022OrphanHuman