Synapse - referralArticle 31 referralsStatus: CHMP opinion Opinion/position date: 21 March 2024Last updated: 6 May 2024ReferralHuman
EU/3/20/2354 - orphan designation for treatment of microvillus inclusion diseaseDate of designation: 13 November 2020PositiveLast updated: 8 April 2024OrphanHuman
Applying for orphan designationLast updated: 19 January 2024HumanRegulatory and procedural guidanceRare diseasesResearch and development
EU/3/10/822 - orphan designation for treatment of Fanconi anaemia type ADate of designation: 17 December 2010PositiveLast updated: 11 September 2023OrphanHuman
EU/3/18/1977 - orphan designation for treatment of fragile X syndromeDate of designation: 22 February 2018PositiveLast updated: 15 June 2023OrphanHuman
EU/3/17/1959 - orphan designation for treatment of tuberous sclerosisDate of designation: 17 January 2018PositiveLast updated: 15 June 2023OrphanHuman
EU/3/17/1921 - orphan designation for treatment of Rett syndromeDate of designation: 16 October 2017PositiveLast updated: 15 June 2023OrphanHuman
EU/3/16/1621 - orphan designation for treatment of gliomaDate of designation: 17 February 2016PositiveLast updated: 15 June 2023OrphanHuman
EU/3/17/1920 - orphan designation for treatment of West syndromeDate of designation: 16 October 2017PositiveLast updated: 15 June 2023OrphanHuman
EU/3/17/1855 - orphan designation for treatment of Lennox-Gastaut syndromeDate of designation: 20 March 2017PositiveLast updated: 14 June 2023OrphanHuman