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235 results
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Summary of opinion: Flynpovi (updated)
eflornithine, sulindac, opinion date: 24/06/2021, Negative, Last updated: 23/07/2021 -
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Summary of opinion: Koselugo
selumetinib, opinion date: 22/04/2021, Positive, Last updated: 23/04/2021 -
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Summary of opinion: Darzalex
daratumumab, opinion date: 20/05/2021, Positive, Last updated: 21/05/2021 -
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Summary of opinion: Galafold
migalastat, opinion date: 24/06/2021, Positive, Last updated: 25/06/2021 -
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Summary of opinion: Voxzogo
vosoritide, opinion date: 24/06/2021, Positive, Last updated: 25/06/2021 -
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Summary of opinion: Minjuvi
tafasitamab, opinion date: 24/06/2021, Positive, Last updated: 25/06/2021 -
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Summary of opinion: Abecma
idecabtagene vicleucel, opinion date: 24/06/2021, Positive, Last updated: 25/06/2021 -
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Summary of opinion: Nexviadyme (new)
Avalglucosidase alfa, opinion date: 23/07/2021, Positive, Last updated: 23/07/2021 -
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Summary of opinion: Deltyba (new)
delamanid, opinion date: 22/07/2021, Positive, Last updated: 23/07/2021 -
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Orphan designation: (2S)-2-{[(2R)-2-[({[3,3-dibutyl-7-(methylthio)-1,1-dioxido-5-phenyl-2,3,4,5-tetrahydro-1,2,5-benzothiadiazepin-8-yl]oxy}acetyl)amino]-2-(4-hydroxyphenyl)acetyl]amino}butanoic acid for: Treatment of progressive familial intrahepatic cholestasis (updated)
Date of designation: 17/07/2012, Positive, Last updated: 28/07/2021 -
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Orphan designation: Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA (elivaldogene autotemcel) for: Treatment of adrenoleukodystrophy (updated)
Date of designation: 06/06/2012, Positive, Last updated: 27/07/2021 -
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Orphan designation: Setmelanotide for: Treatment of pro-opiomelanocortin deficiency (updated)
Date of designation: 14/07/2016, Positive, Last updated: 22/07/2021 -
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Orphan designation: Setmelanotide for: Treatment of leptin receptor deficiency (updated)
Date of designation: 19/11/2018, Positive, Last updated: 22/07/2021 -
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Orphan designation: Treprostinil sodium for: Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension (updated)
Date of designation: 14/04/2004, Positive, Last updated: 16/07/2021 -
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Orphan designation: Carboxypeptidase G2 (glucarpidase) for: Adjunctive treatment in patients at risk of methotrexate toxicity (updated)
Date of designation: 03/02/2003, Positive, Last updated: 06/07/2021 -
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Orphan designation: "Synthetic 47-amino-acid N-myristoylated lipopeptide, derived from the preS region of hepatitis B virus (bulevirtide) for: Treatment of hepatitis delta virus infection
Date of designation: 19/06/2015, Positive, Last updated: 30/06/2021 -
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Orphan designation: Humanised anti-IL-6 receptor monoclonal antibody (satralizumab) for: Treatment of neuromyelitis optica spectrum disorders
Date of designation: 27/06/2016, Positive, Last updated: 30/06/2021 -
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Orphan designation: Pomalidomide for: Treatment of multiple myeloma
Date of designation: 08/10/2009, Positive, Last updated: 10/06/2021 -
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Orphan designation: Ivosidenib for: Treatment of acute myeloid leukaemia
Date of designation: 12/12/2016, Positive, Last updated: 08/06/2021 -
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Orphan designation: Ivacaftor, N-(1,3-dimethyl-1H-pyrazole-4-sulfonyl)-6-[3-(3,3,3-trifluoro-2,2-dimethylpropoxy)-1H-pyrazol-1-yl]-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridine-3-carboxamide, tezacaftor for: Treatment of cystic fibrosis
Date of designation: 14/12/2018, Positive, Last updated: 06/05/2021 -
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Orphan designation: Risdiplam for: Treatment of spinal muscular atrophy
Date of designation: 26/02/2019, Positive, Last updated: 04/05/2021 -
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Orphan designation: pemigatinib for: Treatment of biliary tract cancer
Date of designation: 24/08/2018, Positive, Last updated: 04/05/2021 -
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Orphan designation: Cannabidiol for: Treatment of tuberous sclerosis
Date of designation: 17/01/2018, Positive, Last updated: 26/04/2021 -
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Orphan designation: Somapacitan for: Treatment of growth hormone deficiency
Date of designation: 24/08/2018, Positive, Last updated: 15/04/2021 -
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Orphan designation: Murine anti-CD22 antibody variable region fused to truncated Pseudomonas exotoxin 38 ( Moxetumomab pasudotox) for: Treatment of hairy cell leukaemia
Date of designation: 05/12/2008, Positive, Last updated: 09/03/2021