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Orphan designation: fidanacogene elaparvovec for: Treatment of haemophilia B
Date of designation: 19/11/2018, Positive, Last updated: 19/02/2019 -
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Orphan designation: Rozanolixizumab for: Treatment of myasthenia gravis
Date of designation: 22/04/2020, Positive, Last updated: 13/08/2020 -
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Orphan designation: 4-[(3S)-3-aminopyrrolidin-1-yl]-6-cyano-5-(3,5-difluorophenyl)-N-[(2S)-1,1,1-trifluoropropan-2-yl]pyridine-3-carboxamide for: Treatment of congenital hyperinsulinism
Date of designation: 09/12/2020, Positive, Last updated: 27/05/2021 -
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Orphan designation: perflubron for: Treatment of respiratory distress syndrome
Date of designation: 09/12/2020, Positive, Last updated: 16/09/2021 -
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Orphan designation: Sulindac for: Treatment of fragile X syndrome
Date of designation: 09/12/2020, Positive, Last updated: 12/10/2021 -
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Orphan designation: Navtemadlin for: Treatment of Merkel cell carcinoma
Date of designation: 14/01/2022, Positive, Last updated: 09/06/2022 -
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Orphan designation: Bovactant for: Treatment of respiratory distress syndrome
Date of designation: 24/02/2022, Positive, Last updated: 29/04/2022 -
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Orphan designation: Gadolinium-chelated polysiloxane nanoparticles for: Treatment of pancreatic cancer
Date of designation: 24/02/2022, Positive, Last updated: 29/04/2022 -
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Orphan designation: [Ala1,3,12,Gln10,Arg11,Trp14]PTH(1-14)/[Ala18,22, Lys26]PTHrP(15-36)COOH for: Treatment of hypoparathyroidism
Date of designation: 24/02/2022, Positive, Last updated: 29/04/2022 -
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Orphan designation: aldesleukin for: Treatment of amyotrophic lateral sclerosis
Date of designation: 24/02/2022, Positive, Last updated: 29/04/2022 -
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Orphan designation: Eltanexor for: Treatment of myelodysplastic syndromes
Date of designation: 21/06/2022, Positive, Last updated: 22/09/2022 -
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Orphan designation: Octreotide acetate for: Treatment of carcinoid syndrome
Date of designation: 13/01/2023, Positive, Last updated: 23/03/2023 -
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Orphan designation: Govorestat for: Treatment of galactosaemia (updated)
Date of designation: 21/06/2022, Positive, Last updated: 10/05/2023 -
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Orphan designation: epcoritamab for: Treatment of follicular lymphoma
Date of designation: 21/06/2022, Positive, Last updated: 22/09/2022 -
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Orphan designation: Heterologous human adult liver-derived progenitor cells for: Treatment of argininosuccinic aciduria
Date of designation: 17/07/2013, Positive, -
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Orphan designation: (S)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl)piperazin-yl)pyrimidin-5-yl)ethan-1-amine for: Treatment of gastrointestinal stromal tumours
Date of designation: 17/07/2017, Positive, Last updated: 22/05/2019 -
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Orphan designation: Masitinib mesilate for: Treatment of pancreatic cancer
Date of designation: 28/10/2009, Positive, -
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Orphan designation: Recombinant protein derived from the saliva of the Ornithodoros moubata tick (nomacopan) for: Treatment of Guillain-Barré syndrome
Date of designation: 27/06/2016, Positive, Last updated: 26/01/2022 -
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Orphan designation: Glyceryl tri-(4-phenylbutyrate) for: Treatment of hyperargininaemia
Date of designation: 10/06/2010, Positive, Last updated: 18/11/2022 -
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Orphan designation: 2',3',5'-Tri-O-acetyluridine for: Treatment of 5-fluorouracil overdose
Date of designation: 15/05/2009, Positive, Last updated: 09/06/2009 -
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Orphan designation: 2-[4-Methoxy-3-(2-m-tolyl-ethoxy)-benzoylamino]-indan-2-carboxylic acid for: Treatment of systemic sclerosis
Date of designation: 12/03/2013, Positive, Last updated: 18/09/2020 -
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Orphan designation: Eflornithine for: Treatment of familial adenomatous polyposis (FAP)
Date of designation: 20/09/2010, Positive, Last updated: 29/04/2019 -
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Orphan designation: Propranolol for: Treatment of soft tissue sarcoma
Date of designation: 12/12/2016, Positive, -
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Orphan designation: Mixture of two adeno-associated viral vectors serotype 8 containing the 5'-half sequence of human MYO7A gene and the 3'-half sequence of human MYO7A gene for: Treatment of Usher syndrome
Date of designation: 04/07/2014, Positive, Last updated: 07/11/2022 -
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Orphan designation: polyethylene glycol-modified human recombinant truncated cystathionine beta-synthase (pegtibatinase) for: Treatment of homocystinuria
Date of designation: 30/05/2016, Positive, Last updated: 06/02/2023