Orphan designation: avenciguat Treatment of systemic sclerosis, 11/11/2024 Withdrawn
Submitted by Anonymous (not verified) on 18 May 2026 - 10:38
Orphan designation: avenciguat Treatment of systemic sclerosis, 11/11/2024 Withdrawn
Orphan designation: ataluren Treatment of Becker muscular dystrophy, 04/07/2012 Withdrawn
Submitted by Anonymous (not verified) on 18 May 2026 - 10:37
Orphan designation: ataluren Treatment of Becker muscular dystrophy, 04/07/2012 Withdrawn
Orphan designation: ataluren Treatment of aniridia, 09/10/2015 Withdrawn
Submitted by Anonymous (not verified) on 18 May 2026 - 10:36
Orphan designation: ataluren Treatment of aniridia, 09/10/2015 Withdrawn
Orphan designation: tipifarnib Treatment of peripheral T-cell lymphoma, 19/10/2020 Withdrawn
Submitted by Anonymous (not verified) on 18 May 2026 - 10:36
Orphan designation: tipifarnib Treatment of peripheral T-cell lymphoma, 19/10/2020 Withdrawn
Orphan designation: tipifarnib Treatment of acute myeloid leukaemia, 10/03/2005 Withdrawn
Submitted by Anonymous (not verified) on 18 May 2026 - 10:35
Orphan designation: tipifarnib Treatment of acute myeloid leukaemia, 10/03/2005 Withdrawn
Orphan designation: bemarituzumab Treatment of gastric cancer, 21/08/2024 Withdrawn
Submitted by Anonymous (not verified) on 18 May 2026 - 10:33
Orphan designation: bemarituzumab Treatment of gastric cancer, 21/08/2024 Withdrawn
Orphan designation: 2-[4-Methoxy-3-(2-m-tolyl-ethoxy)-benzoylamino]-indan-2-carboxylic acid Treatment of systemic sclerosis, 12/03/2013 Withdrawn
Submitted by Anonymous (not verified) on 18 May 2026 - 10:32
Orphan designation: 2-[4-Methoxy-3-(2-m-tolyl-ethoxy)-benzoylamino]-indan-2-carboxylic acid Treatment of systemic sclerosis, 12/03/2013 Withdrawn
Orphan designation: autologous adipose tissue-derived mesenchymal stem cells Treatment of thromboangiitis obliterans (Buerger's disease), 20/03/2017 Withdrawn
Submitted by Anonymous (not verified) on 18 May 2026 - 10:30
Orphan designation: autologous adipose tissue-derived mesenchymal stem cells Treatment of thromboangiitis obliterans (Buerger's disease), 20/03/2017 Withdrawn
Orphan designation: nimorazole Treatment of squamous-cell carcinoma of the head and neck in patients undergoing radiotherapy, 23/02/2011 Withdrawn
Submitted by Anonymous (not verified) on 18 May 2026 - 10:29
Orphan designation: nimorazole Treatment of squamous-cell carcinoma of the head and neck in patients undergoing radiotherapy, 23/02/2011 Withdrawn
Orphan designation: Lomitapide Treatment of familial chylomicronaemia, 17/12/2010 Withdrawn
Submitted by Anonymous (not verified) on 18 May 2026 - 10:29
Orphan designation: Lomitapide Treatment of familial chylomicronaemia, 17/12/2010 Withdrawn