2195 results
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Orphan designation: Eculizumab for: Treatment of neuromyelitis optica spectrum disorders (updated)
Date of first decision: 05/08/2013, Positive, Last updated: 04/09/2019 -
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Orphan designation: Allogeneic umbilical cord blood cells treated ex vivo with 16,16-dimethyl prostaglandin E2 for: Treatment of acute myeloid leukaemia (updated)
Date of first decision: 13/05/2011, Withdrawn, Last updated: 30/08/2019 -
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Orphan designation: 2-[[3-({4-[(5-{2-[(3-Fluorophenyl)amino]-2-oxoethyl}-1H-pyrazol-3-yl)amino]-quinazolin-7-yl}oxy)propyl](ethyl)amino]ethyl dihydrogen phosphate trihydrate for: Treatment of acute myeloid leukaemia (updated)
Date of first decision: 05/12/2008, Withdrawn, Last updated: 30/08/2019 -
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Orphan designation: Treosulfan for: Conditioning treatment prior to haematopoietic-progenitor-cell transplantation (updated)
Date of first decision: 22/02/2004, Withdrawn, Last updated: 30/08/2019 -
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Orphan designation: 5'-CTG CCA CGT TCT CCT GC-(2' methoxy)A-(2' methoxy)C-(2' methoxy)C-3' for: Treatment of myasthenia gravis (updated)
Date of first decision: 21/06/2004, Withdrawn, Last updated: 30/08/2019 -
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Orphan designation: Eculizumab for: Treatment of paroxysmal nocturnal haemoglobinuria (updated)
Date of first decision: 17/10/2003, Expired, Last updated: 30/08/2019 -
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Orphan designation: Imetelstat sodium for: Treatment of myelofibrosis (updated)
Date of first decision: 14/12/2015, Positive, Last updated: 23/08/2019 -
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Orphan designation: Lonafarnib for: Treatment of Hutchinson-Gilford progeria
Date of first decision: 14/12/2018, Positive, Last updated: 19/08/2019 -
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Orphan designation: Exendin (9-39) for: Treatment of noninsulinoma pancreatogenous hypoglycaemia syndrome
Date of first decision: 14/10/2016, Positive, Last updated: 19/08/2019 -
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Orphan designation: Ubenimex for: Treatment of pulmonary arterial hypertension
Date of first decision: 21/03/2016, Positive, Last updated: 19/08/2019 -
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Orphan designation: Lonafarnib for: Treatment of hepatitis delta virus infection
Date of first decision: 16/01/2014, Positive, Last updated: 19/08/2019 -
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Orphan designation: Sodium benzoate for: Treatment of hyperargininaemia
Date of first decision: 11/01/2016, Positive, Last updated: 19/08/2019 -
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Orphan designation: Sodium benzoate for: Treatment of argininosuccinic aciduria
Date of first decision: 11/01/2016, Positive, Last updated: 19/08/2019 -
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Orphan designation: Sodium nitrite for: Treatment of aneurysmal subarachnoid haemorrhage
Date of first decision: 16/01/2014, Positive, Last updated: 19/08/2019 -
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Orphan designation: Sodium thiosulfate for: Treatment for calciphylaxis
Date of first decision: 15/01/2015, Positive, Last updated: 19/08/2019 -
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Orphan designation: Diacerein for: Treatment of epidermolysis bullosa
Date of first decision: 29/05/2019, Positive, Last updated: 19/08/2019 -
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Orphan designation: Heat-killed Mycobacterium obuense (whole cell) for: Treatment of pancreatic cancer
Date of first decision: 16/12/2014, Positive, Last updated: 19/08/2019 -
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Orphan designation: Recombinant fragment of human surfactant protein-D for: Prevention of bronchopulmonary dysplasia
Date of first decision: 23/08/2017, Positive, Last updated: 19/08/2019 -
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Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against p53 mRNA (teprasiran) for: Prevention of delayed graft function after renal transplantation
Date of first decision: 06/06/2010, Positive, Last updated: 19/08/2019 -
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Orphan designation: Tamoxifen citrate for: Treatment of Duchenne muscular dystrophy
Date of first decision: 12/10/2017, Positive, Last updated: 30/07/2019 -
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Orphan designation: Metformin and L-citrulline for: Treatment of Duchenne muscular dystrophy
Date of first decision: 17/01/2018, Positive, Last updated: 30/07/2019 -
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Orphan designation: Adeno-associated viral vector serotype 2/6 encoding zinc-finger nucleases and the human alpha L-iduronidase gene for: Treatment of mucopolysaccharidosis type I
Date of first decision: 17/01/2018, Positive, Last updated: 30/07/2019 -
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Orphan designation: Adeno-associated viral vector serotype 2/6 encoding zinc-finger nucleases and the human iduronate 2-sulfatase gene for: Treatment of mucopolysaccharidosis type II (Hunter's syndrome)
Date of first decision: 17/01/2018, Positive, Last updated: 30/07/2019 -
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Orphan designation: Recombinant adeno-associated viral vector serotype 6 encoding the B-domain-deleted human factor VIII for: Treatment of haemophilia A
Date of first decision: 22/05/2017, Positive, Last updated: 30/07/2019 -
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Orphan designation: trientine dihydrochloride for: Treatment of Wilson's disease
Date of first decision: 24/10/2003, Withdrawn, Last updated: 29/07/2019