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Orphan designation: Recombinant human acid sphingomyelinase (also known as olipudase alfa) for: Treatment of Niemann-Pick disease
Date of designation: 24/06/2022, Positive, Last updated: 27/07/2022 -
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Orphan designation: Adeno-associated viral vector serotype 9 containing the human SMN gene (onasemnogene abeparvovec) for: Treatment of spinal muscular atrophy
Date of designation: 19/06/2015, Positive, Last updated: 25/07/2022 -
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Orphan designation: Autologous CD34+ cells transfected with lentiviral vector containing the human arylsulfatase A cDNA for: treatment of metachromatic leukodystrophy
Date of designation: 13/04/2007, Positive, Last updated: 14/07/2022 -
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Orphan designation: Autologous CD34+ cells transfected with retroviral vector containing adenosine deaminase gene for: Treatment of severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency
Date of designation: 26/08/2005, Positive, Last updated: 14/07/2022 -
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Orphan designation: Autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor (axicabtagene ciloleucel) for: Treatment of follicular lymphoma
Date of designation: 11/11/2015, Positive, Last updated: 12/07/2022 -
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Orphan designation: dry extract from birch bark (DER 5-10 : 1), extraction solvent n-heptane 95% (w/w) (birch bark extract) for: Treatment of epidermolysis bullosa
Date of designation: 23/02/2011, Positive, Last updated: 04/07/2022 -
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Orphan designation: (2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4-methyl-3-trifluoromethylphenyl)amNide for: Treatment of microscopic polyangiitis (avacopan)
Date of designation: 19/11/2014, Positive, Last updated: 04/07/2022 -
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Orphan designation: (2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4-methyl-3-trifluoromethylphenyl)amide (avacopan) for: Treatment of granulomatosis with polyangiitis
Date of designation: 19/11/2014, Positive, Last updated: 04/07/2022 -
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Orphan designation: Setmelanotide for: Treatment of leptin receptor deficiency
Date of designation: 19/11/2018, Positive, Last updated: 01/07/2022 -
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Orphan designation: Setmelanotide for: Treatment of pro-opiomelanocortin deficiency
Date of designation: 14/07/2016, Positive, Last updated: 01/07/2022 -
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Orphan designation: Mosunetuzumab for: Treatment of follicular lymphoma
Date of designation: 12/11/2021, Positive, Last updated: 23/06/2022 -
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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
Date of designation: 28/02/2020, Positive, Last updated: 13/06/2022 -
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Orphan designation: polatuzumab vedotin for: Treatment of diffuse large B-cell lymphoma
Date of designation: 16/04/2018, Positive, Last updated: 08/06/2022 -
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Orphan designation: tisagenlecleucel for: Treatment of follicular lymphoma
Date of designation: 19/07/2021, Positive, Last updated: 19/05/2022 -
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Orphan designation: Tebentafusp for: Treatment of uveal melanoma
Date of designation: 19/02/2021, Positive, Last updated: 22/04/2022 -
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Orphan designation: mifepristone for: Treatment of hypercortisolism (Cushing's syndrome) of endogenous origin
Date of designation: 27/10/2011, Positive, Last updated: 18/03/2022 -
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Orphan designation: 2-hydroxy-6-((2-(1-isopropyl-1H-pyrazol-5-yl)pyridin-3-yl)methoxy)benzaldehyde (voxelotor) for: Treatment of sickle cell disease
Date of designation: 18/11/2016, Positive, Last updated: 17/03/2022 -
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Orphan designation: Recombinant modified human growth hormone for: Treatment of growth hormone deficiency
Date of designation: 24/01/2013, Positive, Last updated: 03/03/2022 -
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Orphan designation: Donor T lymphocytes depleted ex vivo of host alloreactive T cells using photodynamic treatment- for: Treatment in haematopoietic stem cell transplantation
Date of designation: 27/06/2016, Withdrawn, Last updated: 28/02/2022 -
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Orphan designation: Donor T lymphocytes depleted ex-vivo of host alloreactive T cells using photodynamic treatment for: Treatment of acute myeloid leukaemia
Date of designation: 19/11/2014, Withdrawn, Last updated: 28/02/2022 -
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Orphan designation: Donor lymphocyte preparation depleted of functional alloreactive T-cells for: Prevention of graft-versus-host disease
Date of designation: 05/09/2008, Withdrawn, Last updated: 28/02/2022 -
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Orphan designation: Octreotide acetate for: Treatment of acromegaly
Date of designation: 05/08/2013, Positive, Last updated: 21/02/2022 -
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Orphan designation: lonapegsomatropin for: Treatment of growth hormone deficiency
Date of designation: 17/10/2019, Positive, Last updated: 17/02/2022 -
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Orphan designation: [5-(5-chloro-1H-pyrrolo[2,3-b]pyridin-3-ylmethyl)-pyridin-2-yl]-(6-trifluoromethyl-pyridin-3-ylmethyl)-amine hydrochloride for: Treatment of tenosynovial giant cell tumour, localised and diffuse type
Date of designation: 19/03/2015, Withdrawn, Last updated: 02/02/2022 -
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Orphan designation: ivacaftor, tezacaftor for: Treatment of cystic fibrosis
Date of designation: 27/02/2017, Positive, Last updated: 25/01/2022