For help on how to get the results you want, see our search tips.
4143 results
Medicine
Orphan designations Remove Orphan designations filter
Periodic safety update report single assessments Remove Periodic safety update report single assessments filter
Categories
Human Remove Human filter
-
List item
Orphan designation: (1S,3S)-3-amino-4-(difluoromethylene) cyclopentanecarboxylic acid hydrochloride for: Treatment of West syndrome
Date of first decision: 09/02/2012, Withdrawn, Last updated: 03/02/2021 -
List item
Orphan designation: Rovalpituzumab tesirine for: Treatment of small cell lung cancer
Date of first decision: 30/05/2016, Withdrawn, Last updated: 03/02/2021 -
List item
Orphan designation: Oxalobacter formigenes strain HC-1 for: Treatment of primary hyperoxaluria
Date of first decision: 17/02/2006, Positive, Last updated: 02/02/2021 -
List item
Orphan designation: Oxalobacter formigenes strain HC-1 for: Treatment of short bowel syndrome
Date of first decision: 15/10/2014, Positive, Last updated: 02/02/2021 -
List item
Orphan designation: Somapacitan for: Treatment of growth hormone deficiency
Date of first decision: 24/08/2018, Positive, Last updated: 01/02/2021 -
List item
Orphan designation: pemigatinib for: Treatment of myeloid/lymphoid neoplasms with eosinophilia and rearrangement of PDGFRA, PDGFRB, or FGFR1, or with PCM1-JAK
Date of first decision: 17/10/2019, Positive, Last updated: 01/02/2021 -
List item
Orphan designation: Clofazimine for: Treatment of nontuberculous mycobacterial lung disease
Date of first decision: 21/08/2019, Positive, Last updated: 28/01/2021 -
List item
Orphan designation: Autologous peripheral blood T cells CD4 and CD8 selected and CD3 and CD28 activated transduced with retroviral vector expressing anti CD19 CD28/CD3-zeta chimeric antigen receptor and cultured (KTE-X19) for: Treatment of mantle cell lymphoma
Date of first decision: 13/11/2019, Positive, Last updated: 25/01/2021 -
List item
Orphan designation: recombinant human interleukin-3 truncated diphtheria toxin fusion protein for: Treatment of blastic plasmacytoid dendritic cell neoplasm
Date of first decision: 11/11/2015, Positive, Last updated: 25/01/2021 -
List item
Orphan designation: Oxytocin for: Treatment of Prader-Willi syndrome
Date of first decision: 29/07/2014, Positive, Last updated: 25/01/2021 -
List item
Orphan designation: dimethyl fumarate for: Adjunctive treatment in haematopoietic cell transplantation
Date of first decision: 21/03/2018, Positive, Last updated: 15/01/2021 -
List item
Orphan designation: rasagiline for: Treatment of Duchenne muscular dystrophy
Date of first decision: 28/06/2019, Withdrawn, Last updated: 12/01/2021 -
List item
Orphan designation: Tetrahydrobiopterin for: Treatment of hyperphenylalaninaemia
Date of first decision: 08/06/2004, Withdrawn, Last updated: 11/01/2021 -
List item
Orphan designation: Fenfluramine hydrochloride for: Treatment of Dravet syndrome
Date of first decision: 18/12/2013, Positive, Last updated: 08/01/2021 -
List item
Orphan designation: adeno-associated viral vector serotype 2 containing the human REP1 gene (timrepigene emparvovec) for: Treatment of spinal muscular atrophy
Date of first decision: 24/08/2018, Withdrawn, Last updated: 07/01/2021 -
List item
Orphan designation: Autologous CD34+ cells transfected with lentiviral vector containing the human arylsulfatase A cDNA for: treatment of metachromatic leukodystrophy
Date of first decision: 13/04/2007, Positive, Last updated: 22/12/2020 -
List item
Orphan designation: Miransertib for: Treatment of Proteus syndrome
Date of first decision: 21/03/2018, Positive, Last updated: 22/12/2020 -
List item
Orphan designation: Obeticholic acid for: Treatment of primary sclerosing cholangitis
Date of first decision: 16/03/2014, Positive, Last updated: 22/12/2020 -
List item
Orphan designation: Ibutamoren mesilate for: Treatment of growth hormone deficiency
Date of first decision: 20/06/2017, Positive, Last updated: 22/12/2020 -
List item
Orphan designation: Bardoxolone methyl for: Treatment of Alport syndrome
Date of first decision: 25/05/2018, Positive, Last updated: 21/12/2020 -
List item
Orphan designation: Antisense oligonucleotide targeting the F508delta mutation of CFTR (eluforsen) for: Treatment of cystic fibrosis
Date of first decision: 07/10/2013, Withdrawn, Last updated: 18/12/2020 -
List item
Orphan designation: 1-(6-benzothiazolylsulfonyl)-5-chloro-1H-indole-2-butanoic acid (lanifibranor) for: Treatment of systemic sclerosis
Date of first decision: 19/11/2014, Withdrawn, Last updated: 14/12/2020 -
List item
Orphan designation: 5-amino-1-(2-methyl-1H-benzo[d]imidazol-5-yl)-1H-pyrazol-4-yl 1H-indol-2-yl ketone mono[(S)-2-hydroxysuccinate] for: Treatment of biliary tract cancer
Date of first decision: 16/10/2017, Withdrawn, Last updated: 11/12/2020 -
List item
Orphan designation: Recombinant antibody derivative against human CD19 and CD3 (blinatumomab) for: Treatment of chronic lymphocytic leukaemia
Date of first decision: 01/12/2003, Withdrawn, Last updated: 11/12/2020 -
List item
Orphan designation: Recombinant antibody derivative against human CD19 and CD3 (blinatumomab) for: Treatment of mantle cell lymphoma
Date of first decision: 01/12/2003, Withdrawn, Last updated: 11/12/2020