93 results
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Orphan designation: Mexiletine hydrochloride for: Treatment of non-dystrophic myotonia
Date of designation: 07/06/2013, Positive, Last updated: 30/04/2021 -
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Orphan designation: N-methyl-4-({4-[({3-methyl(methylsulfonyl)aminopyrazin-2-yl}methyl)amino]-5-(trifluoromethyl)pyrimidin-2-yl}amino)benzamide hydrochloride (defactinib) for: Treatment of ovarian cancer
Date of designation: 15/01/2015, Positive, Last updated: 24/05/2022 -
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Orphan designation: (1S,3S)-3-amino-4-(difluoromethylene) cyclopentanecarboxylic acid hydrochloride for: Treatment of West syndrome
Date of designation: 09/02/2012, Withdrawn, Last updated: 03/02/2021 -
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Orphan designation: N-methyl-4-({4-[({3-methyl(methylsulfonyl)aminopyrazin-2-yl}methyl)amino]-5-(trifluoromethyl)pyrimidin-2-yl}amino)benzamide hydrochloride (defactinib) for: Treatment of malignant mesothelioma
Date of designation: 07/06/2013, Withdrawn, Last updated: 28/02/2022 -
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Orphan designation: Ambroxol hydrochloride for: Treatment of amyotrophic lateral sclerosis
Date of designation: 25/05/2018, Positive, Last updated: 24/07/2018 -
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Orphan designation: Forodesine hydrochloride for: Treatment of cutaneous T-cell lymphoma
Date of designation: 29/01/2007, Positive, Last updated: 21/05/2019 -
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Orphan designation: propranolol hydrochloride for: Treatment of von Hippel-Lindau disease
Date of designation: 27/02/2017, Positive, Last updated: 31/03/2017kezelése Italian Propranololo cloridrato Trattamento della sindrome … Hippel-Lindau’a Portuguese Cloridrato propranolol Tratamento da … -
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Orphan designation: Forodesine hydrochloride for: Treatment of acute lymphoblastic leukaemia
Date of designation: 18/12/2006, Positive, Last updated: 21/05/2019 -
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Orphan designation: N-[2,6-bis(1-methylethyl)phenyl]-N'-[[1-[4-(dimethylamino)phenyl]cyclopentyl]methyl]urea, hydrochloride salt for: Treatment of congenital adrenal hyperplasia
Date of designation: 17/01/2018, Positive, Last updated: 26/04/2019 -
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Orphan designation: Acebutolol hydrochloride for: Treatment of Smith-Magenis syndrome
Date of designation: 14/10/2016, Positive, Last updated: 18/03/2019 -
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Orphan designation: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride for: Treatment of adrenoleukodystrophy
Date of designation: 18/11/2016, Positive, Last updated: 13/12/2016 -
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Orphan designation: 3-(5-Amino-2-methyl-4-oxoquinazolin-3(4H)-yl)piperidine-2,6-dione hydrochloride for: Treatment of diffuse large B-cell lymphoma
Date of designation: 27/06/2016, Positive, Last updated: 23/01/2019 -
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Orphan designation: doxorubicin hydrochloride for: Treatment of hepatocellular carcinoma
Date of designation: 23/02/2011, Positive, Last updated: 22/01/2019 -
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Orphan designation: Mexiletine hydrochloride for: Treatment of myotonic disorders
Date of designation: 19/11/2014, Positive, Last updated: 13/06/2018kezelése Italian Mexiletina cloridrato Trattamento dei disordini … miotonicznych Portuguese Cloridrato de mexiletina Tratamento … -
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Orphan designation: 2-amino-2-[2-[2-chloro-4-[[3-(phenylmethoxy)phenyl]thio]phenyl]ethyl]-1,3-propanediol hydrochloride for: Prevention of graft-versus-host disease
Date of designation: 14/12/2015, Withdrawn, Last updated: 22/01/2018 -
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Orphan designation: 5-aminolevulinic acid hydrochloride for: Intra-operative photodynamic diagnosis of residual glioma
Date of designation: 14/11/2002, Expired, Last updated: 03/10/2017 -
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Orphan designation: N-[2,6-bis(1-methylethyl)phenyl]-N'-[[1-[4-(dimethylamino) phenyl]cyclopentyl]methyl]urea, hydrochloride salt for: Treatment of adrenocortical carcinoma
Date of designation: 07/06/2013, Positive, Last updated: 13/11/2017 -
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Orphan designation: Desipramine chlorhydrate for: Treatment of Rett syndrome
Date of designation: 12/06/2009, Withdrawn, Last updated: 16/08/2017kezelése Italian Desipramina cloridrato Trattamento della sindrome … zespołu Retta Portuguese Cloridrato de desipramina Tratamento … -
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Orphan designation: Oxymetazoline hydrochloride for: Treatment of spinal cord injury
Date of designation: 17/07/2017, Positive, Last updated: 15/08/2017 -
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Orphan designation: pioglitazone hydrochloride for: Treatment of sudden sensorineural hearing loss
Date of designation: 12/01/2017, Positive, Last updated: 11/07/2017kezelése Italian Pioglitazone cloridrato Trattamento dell'ipoacusia … -
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Orphan designation: Doxorubicin hydrochloride in a lipid-based pegylated nanoparticle modified with a 31-aminoacid peptide targeting nucleolin for: Treatment of malignant mesothelioma
Date of designation: 12/01/2017, Positive, Last updated: 14/07/2017 -
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Orphan designation: Irinotecan hydrochloride for: Treatment of glioma
Date of designation: 30/11/2007, Withdrawn, Last updated: 25/04/2017 -
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Orphan designation: doxorubicin hydrochloride for: Treatment of glioma
Date of designation: 30/11/2007, Withdrawn, Last updated: 25/04/2017kezelése Italian Doxorubicina cloridrato (microsfere ad eluizione … -
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Orphan designation: N-{2-Chloro-4-[(6,7-dimethoxy-4-quinolyl)oxy]phenyl}-N'-(5-methyl-3-isoxazolyl) urea hydrochloride monohydrate for: Treatment of renal-cell carcinoma
Date of designation: 09/06/2010, Withdrawn, Last updated: 17/05/2017 -
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Orphan designation: Anagrelide hydrochloride for: Treatment of essential thrombocythaemia
Date of designation: 30/12/2000, Expired, Last updated: 08/02/2017