For help on how to get the results you want, see our search tips.
273 results
Medicine
Orphan designations Remove Orphan designations filter
Summaries of opinion Remove Summaries of opinion filter
Categories
Human Remove Human filter
-
List item
Orphan designation: Sirolimus for: Treatment of chronic non-infectious uveitis
Date of designation: 30/08/2011, Withdrawn, Last updated: 30/08/2022 -
List item
Orphan designation: recombinant adeno-associated viral vector serotype 2 carrying the gene for the human aromatic L-amino acid decarboxylase protein, Eladocagene exuparvovec for: Treatment of aromatic L-amino acid decarboxylase deficiency
Date of designation: 18/11/2016, Positive, Last updated: 10/08/2022 -
List item
Orphan designation: (S)-ethyl 2-amino-3-(4-(2-amino-6((R)-1-(4-chloro-2-(3-methyl-1H-pyrazol-1-yl)phenyl)-2,2,2-trifluoroethoxy)pyrimidin-4-yl)phenyl)propanoate for: Treatment of carcinoid syndrome
Date of designation: 30/05/2016, Positive, Last updated: 28/07/2022 -
List item
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 -
List item
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 -
List item
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 -
List item
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 -
List item
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 -
List item
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 -
List item
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 -
List item
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 -
List item
Orphan designation: Setmelanotide for: Treatment of leptin receptor deficiency
Date of designation: 19/11/2018, Positive, Last updated: 01/07/2022 -
List item
Orphan designation: Setmelanotide for: Treatment of pro-opiomelanocortin deficiency
Date of designation: 14/07/2016, Positive, Last updated: 01/07/2022 -
List item
Orphan designation: Mosunetuzumab for: Treatment of follicular lymphoma
Date of designation: 12/11/2021, Positive, Last updated: 23/06/2022 -
List item
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 -
List item
Orphan designation: polatuzumab vedotin for: Treatment of diffuse large B-cell lymphoma
Date of designation: 16/04/2018, Positive, Last updated: 08/06/2022 -
List item
Orphan designation: tisagenlecleucel for: Treatment of follicular lymphoma
Date of designation: 19/07/2021, Positive, Last updated: 19/05/2022 -
List item
Orphan designation: Tebentafusp for: Treatment of uveal melanoma
Date of designation: 19/02/2021, Positive, Last updated: 22/04/2022 -
List item
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 -
List item
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 -
List item
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 -
List item
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 -
List item
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 -
List item
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 -
List item
Orphan designation: Octreotide acetate for: Treatment of acromegaly
Date of designation: 05/08/2013, Positive, Last updated: 21/02/2022