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74 results
Orphan designation status
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Orphan designation: everolimus for: Treatment of tuberous sclerosis
Date of designation: 04/08/2010, Expired, Last updated: 15/09/2023 -
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Orphan designation: Pomalidomide for: Treatment of multiple myeloma
Date of designation: 08/10/2009, Expired, Last updated: 13/09/2023 -
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Orphan designation: Tobramycin for: Treatment of Pseudomonas aeruginosa lung infection in cystic fibrosis
Date of designation: 17/03/2003, Expired, Last updated: 31/07/2023 -
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Orphan designation: Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl] (ponatinib) for: Treatment of acute lymphoblastic leukaemia
Date of designation: 02/02/2010, Expired, Last updated: 20/07/2023 -
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Orphan designation: Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl] (ponatinib) for: Treatment of chronic myeloid leukaemia
Date of designation: 02/02/2010, Expired, Last updated: 20/07/2023 -
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Orphan designation: proteolytic enzymes enriched in bromelain for: Treatment of partial deep dermal and full-thickness burns
Date of designation: 30/07/2002, Expired, Last updated: 15/02/2023 -
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Orphan designation: velaglucerase alfa for: Treatment of Gaucher disease
Date of designation: 06/06/2010, Expired, Last updated: 09/09/2022 -
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Orphan designation: N-(2,4-di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3-carboxamide (ivacaftor) for: Treatment of cystic fibrosis
Date of designation: 08/07/2008, Expired, Last updated: 10/08/2022 -
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Orphan designation: pasireotide for: Treatment of Cushing's disease
Date of designation: 08/10/2009, Expired, Last updated: 14/06/2022 -
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Orphan designation: Mannitolum for: Treatment of cystic fibrosis
Date of designation: 07/11/2005, Expired, Last updated: 05/05/2022 -
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Orphan designation: Mercaptopurine for: Treatment of acute lymphoblastic leukaemia
Date of designation: 30/04/2009, Expired, Last updated: 05/05/2022 -
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Orphan designation: N-methyl D-(2,3,4,5,6-pentahydroxy-hexyl)-ammonium; 2-(3,5-dichloro-phenyl)-benzoxazole-6-carboxylate (tafamidis) for: Treatment of familial amyloid polyneuropathy
Date of designation: 28/08/2006, Expired, Last updated: 25/11/2021 -
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Orphan designation: hydrocortisone for: Treatment of adrenal insufficiency
Date of designation: 22/05/2006, Expired, Last updated: 25/11/2021 -
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Orphan designation: 1, 1'-[1,4-Phenylenebis (methylene)]-bis-1,4,8,11-tetraazacyclotetradecane (plerixafor) for: Treatment to mobilize progenitor cells prior to stem cell transplantation
Date of designation: 20/10/2004, Expired, Last updated: 10/08/2021 -
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Orphan designation: carglumic acid for: Treatment of propionic acidaemia
Date of designation: 07/11/2008, Expired, Last updated: 09/06/2021 -
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Orphan designation: carglumic acid for: Treatment of isovaleric acidaemia
Date of designation: 07/11/2008, Expired, Last updated: 09/06/2021 -
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Orphan designation: carglumic acid for: Treatment of methylmalonic acidaemia
Date of designation: 07/11/2008, Expired, Last updated: 09/06/2021 -
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Orphan designation: Pirfenidone for: Treatment of idiopathic pulmonary fibrosis
Date of designation: 16/11/2004, Expired, Last updated: 22/03/2021 -
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Orphan designation: icatibant acetate for: Treatment of angioedema
Date of designation: 18/02/2003, Expired, Last updated: 12/08/2020 -
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Orphan designation: nilotinib for: Treatment of chronic myeloid leukaemia
Date of designation: 22/05/2006, Expired, Last updated: 20/01/2020 -
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Orphan designation: trabectedin for: Treatment of ovarian cancer
Date of designation: 17/10/2003, Expired, Last updated: 27/11/2019 -
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Orphan designation: Eculizumab for: Treatment of paroxysmal nocturnal haemoglobinuria
Date of designation: 17/10/2003, Expired, Last updated: 20/11/2019 -
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Orphan designation: Aztreonam lysinate for: Treatment of Gram-negative bacterial lung infection in cystic fibrosis
Date of designation: 21/06/2004, Expired, Last updated: 16/10/2019 -
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Orphan designation: Muramyl tripeptide phosphatidyl ethanolamine for: Treatment of osteosarcoma
Date of designation: 21/06/2004, Expired, Last updated: 11/07/2019 -
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Orphan designation: Caffeine citrate for: Treatment of primary apnoea of premature newborns
Date of designation: 18/02/2003, Expired, Last updated: 11/07/2019