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Orphan designation: Mosunetuzumab for: Treatment of follicular lymphoma (updated)
Date of designation: 12/11/2021, Positive, Last updated: 23/06/2022 -
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Orphan designation: Chimeric locked nucleic acid deoxynucleoside phosphorothioate-linked oligonucleotide inhibitor directed against microRNA-155-5p for: Treatment of cutaneous T-cell lymphoma (updated)
Date of designation: 22/05/2017, Withdrawn, Last updated: 22/06/2022 -
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Orphan designation: Protein-based delivery vector carrying a DNA payload encoding an RNA-guided nuclease that targets stx genes of Shiga toxin-producing Escherichia coli for: Prevention of haemolytic uraemic syndrome (updated)
Date of designation: 21/08/2020, Positive, Last updated: 22/06/2022 -
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Orphan designation: Modified messenger ribonucleic acid encoding human propionyl-coenzyme A carboxylase alpha and beta subunits encapsulated into lipid nanoparticle for: Treatment of propionic acidaemia (updated)
Date of designation: 24/04/2019, Positive, Last updated: 22/06/2022 -
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Orphan designation: Modified mRNA encoding human methylmalonyl-coenzyme A mutase encapsulated into lipid nanoparticles for: Treatment of methylmalonic acidaemia (updated)
Date of designation: 25/05/2018, Positive, Last updated: 22/06/2022 -
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Orphan designation: Synthetic signal peptide of human mucin-1 (amino acids 1-21) for: Treatment of plasma cell myeloma (updated)
Date of designation: 15/01/2015, Positive, Last updated: 22/06/2022 -
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Orphan designation: Methyl 4,6-diamino-2-[1-(2-fluorobenzyl)-1H-pyrazolo[3,4-b]pyridine-3-yl]-5-pyrimidinyl(methyl)carbamate (riociguat) for: Treatment of pulmonary arterial hypertension (updated)
Date of designation: 20/12/2007, Withdrawn, Last updated: 14/06/2022 -
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Orphan designation: (4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5- tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyclo[2.2.2]octane chloride (maralixibat chloride) for: Treatment of primary sclerosing cholangitis (updated)
Date of designation: 16/01/2014, Withdrawn, Last updated: 14/06/2022 -
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Orphan designation: (4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5- tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyclo[2.2.2]octane chloride (maralixibat chloride) for: Treatment of primary biliary cirrhosis (updated)
Date of designation: 16/01/2014, Withdrawn, Last updated: 14/06/2022 -
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Orphan designation: Human autologous mesenchymal adult stem cells extracted from adipose tissue for: Treatment of anal fistula (updated)
Date of designation: 26/08/2005, Positive, Last updated: 14/06/2022 -
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Orphan designation: Adeno-associated viral vector serotype rh10 containing the human cholesterol 24-hydroxylase gene for: Treatment of Huntington’s disease (updated)
Date of designation: 01/04/2019, Positive, Last updated: 14/06/2022 -
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Orphan designation: Adeno-associated viral vector serotype 8 containing the human acid alpha-glucosidase gene for: Treatment of glycogen storage disease type II (Pompe's disease) (updated)
Date of designation: 16/04/2018, Positive, Last updated: 14/06/2022 -
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Orphan designation: pasireotide for: Treatment of Cushing's disease (updated)
Date of designation: 08/10/2009, Expired, Last updated: 14/06/2022 -
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Orphan designation: Autologous human T cells genetically modified ex-vivo with a lentiviral vector encoding a chimeric antigen receptor for B-cell maturation antigen for: Treatment of multiple myeloma (updated)
Date of designation: 28/02/2020, Positive, Last updated: 13/06/2022 -
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Orphan designation: Refusal of orphan designation for the treatment of uveal melanoma (updated)
Date of refusal of designation: 11/01/2022, Negative, Last updated: 09/06/2022 -
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Orphan designation: Mecasermin rinfabate for: Prevention of retinopathy of prematurity in neonates of less than 32 weeks of gestational age (updated)
Date of designation: 28/08/2006, Positive, Last updated: 08/06/2022 -
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Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding galactosidase alpha for: Treatment of Fabry disease (updated)
Date of designation: 19/10/2020, Positive, Last updated: 08/06/2022 -
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Orphan designation: Autologous CD34+ cells transfected with a lentiviral vector containing codon optimised RPS19 gene for: Treatment of Diamond-Blackfan anaemia (updated)
Date of designation: 20/08/2021, Positive, Last updated: 08/06/2022 -
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Orphan designation: polatuzumab vedotin for: Treatment of diffuse large B-cell lymphoma (updated)
Date of designation: 16/04/2018, Positive, Last updated: 08/06/2022 -
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Orphan designation: Trifarotene for: Treatment of autosomal recessive congenital ichthyosis (updated)
Date of designation: 24/03/2020, Withdrawn, Last updated: 07/06/2022 -
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Orphan designation: Allogeneic retinal pigment epithelial cells genetically modified with a non-viral vector to express human alpha-L-iduronidase for: Treatment of mucopolysaccharidosis type I
Date of designation: 15/10/2021, Positive, Last updated: 24/05/2022 -
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Orphan designation: Allogeneic retinal pigment epithelial cells genetically modified with a non-viral vector to express beta-domain deleted human factor VIII for: Treatment of haemophilia A
Date of designation: 09/12/2020, Positive, Last updated: 24/05/2022 -
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Orphan designation: Tipifarnib for: Treatment of peripheral T-cell lymphoma
Date of designation: 19/10/2020, Positive, Last updated: 24/05/2022 -
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Orphan designation: 6-((3S,4S)-4-methyl-1-(pyrimidin-2-yl-methyl)pyrrolidin-3-yl)-3-tetrahydropyran-4-yl-7H-imidazo(1,5-a)pyrazin-8-one for: Treatment of sickle cell disease
Date of designation: 21/08/2020, Positive, Last updated: 24/05/2022 -
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Orphan designation: 3-(3-(3,5-bis(trifluoromethyl)phenyl)-1h-pyrazol-1-yl)propanoic acid for: Treatment of Stargardt's disease
Date of designation: 24/04/2019, Positive, Last updated: 24/05/2022