For help on how to get the results you want, see our search tips.
4503 results
-
List item
Orphan designation: Brivanib alaninate for: Treatment of hepatocellular carcinoma
Date of first decision: 27/10/2011, Withdrawn, -
List item
Orphan designation: (Z)-N-[2-(Diethylamino)ethyl]-5-[(5-fluoro-2-oxo-1,2-dihydro-3H-indol-3-ylidene)methyl]-2,4-dimethyl-1H-pyrrole-3-carboxamide (S)-2-hydroxysyccinate for: Treatment of malignant gastrointestinal stromal tumours
Date of first decision: 10/03/2005, Withdrawn, Last updated: 15/10/2009 -
List item
Orphan designation: Tralokinumab for: Treatment of idiopathic pulmonary fibrosis
Date of first decision: 08/11/2012, Withdrawn, -
List item
Orphan designation: Antisense oligonucleotide (TATCCGGAGGGCTCGCCATGCTGCT) for: Treatment of retinopathy of prematurity
Date of first decision: 02/10/2003, Positive, -
List item
Orphan designation: Talarozole for: Treatment of autosomal recessive congenital ichthyosis
Date of first decision: 04/07/2012, Withdrawn, Last updated: 19/01/2016 -
List item
Orphan designation: Recombinant human placental growth factor for: Treatment of pre-eclampsia
Date of first decision: 27/06/2018, Positive, -
List item
Orphan designation: Monoclonal antibody to human interleukin-6 for: Treatment of post-transplantation lymphoproliferative disorders
Date of first decision: 18/12/2002, Withdrawn, -
List item
Orphan designation: 4-[2-(6-Methylpyridin-2-yl)-5,6-dihydro-4H-pyrrolo[1,2-b]pyrazol-3-yl]-quinoline-6-carboxamide monohydrate for: Treatment of glioma
Date of first decision: 26/04/2013, Withdrawn, Last updated: 17/06/2019 -
List item
Orphan designation: Soluble recombinant human fibroblast growth factor receptor 3 for: Treatment of achondroplasia
Date of first decision: 27/02/2017, Positive, Last updated: 31/03/2017 -
List item
Orphan designation: 1,3-Propanedisulfonic acid, disodium salt for: Treatment of systemic secondary amyloidosis
Date of first decision: 31/07/2001, Positive, -
List item
Orphan designation: 20-pentaerythritol poly (oxy-1,2-ethanediyl)-carboxymethyl-glycinate-7-ethyl-10-hydroxycamptothecine 10-[1,4'-bipiperidine]-1'-carboxylate for: Treatment of ovarian cancer
Date of first decision: 27/09/2011, Positive, -
List item
Orphan designation: Autologous urothelial and smooth muscle cells for: Treatment of spina bifida
Date of first decision: 18/03/2008, Withdrawn, Last updated: 24/05/2012 -
List item
Orphan designation: Sodium hypochlorite for: Treatment of partial deep dermal and full-thickness burns
Date of first decision: 14/07/2016, Positive, Last updated: 06/09/2016 -
List item
Orphan designation: Humanised anti-IL-6 receptor monoclonal antibody (satralizumab) for: Treatment of neuromyelitis optica spectrum disorders
Date of first decision: 27/06/2016, Positive, Last updated: 19/09/2019 -
List item
Orphan designation: Polyethylene glycol-modified human recombinant truncated cystathionine beta-synthase for: Treatment of homocystinuria
Date of first decision: 30/05/2016, Positive, Last updated: 23/06/2016 -
List item
Orphan designation: Allogeneic motor neuron progenitor cells derived from human embryonic stem cells for: Treatment of 5q spinal muscular atrophy
Date of first decision: 24/01/2013, Positive, Last updated: 01/03/2013 -
List item
Orphan designation: Amrubicin hydrochloride for: Treatment of small cell lung cancer
Date of first decision: 02/04/2008, Withdrawn, Last updated: 11/08/2016 -
List item
Orphan designation: Allogeneic and autologous haptenised and irradiated cells and cell lysates derived from glioma for: Treatment of glioma
Date of first decision: 16/01/2014, Positive, -
List item
Orphan designation: 3-pentylbenzeneacetic acid sodium salt for: Treatment of Alström syndrome
Date of first decision: 12/01/2017, Positive, Last updated: 05/06/2019 -
List item
Orphan designation: Sapacitabine for: Treatment of myelodysplastic syndromes
Date of first decision: 08/07/2008, Positive, Last updated: 15/05/2009 -
List item
Orphan designation: Cannabidiol for: Treatment of tuberous sclerosis
Date of first decision: 17/01/2018, Positive, Last updated: 23/04/2019 -
List item
Orphan designation: Adeno-associated viral vector serotype 8 encoding engineered rhodopsin DNA-binding repressor and human rhodopsin expression cassettes for: Treatment of retinitis pigmentosa
Date of first decision: 12/12/2016, Positive, -
List item
Orphan designation: afamelanotide for: Treatment of solar urticaria
Date of first decision: 24/07/2009, Positive, Last updated: 11/04/2016 -
List item
Orphan designation: Recombinant human elafin for: Treatment of oesophagus carcinoma
Date of first decision: 28/01/2010, Positive, Last updated: 25/02/2010 -
List item
Orphan designation: [N-((2S,3R,3aS,3'R,4a'R,6S,6a'R,6b'S,7aR,12a'S,12b'S,Z)-3,6,11',12b'-tetramethyl-2',3a,3',4,4',4a',5,5',6,6',6a',6b',7,7a,7',8',10',12',12a',12b'-icosahydro-1'H,3H-spiro[furo[3,2-b]pyridine-2,9'-naphtho[2,1-a]azulene]-3'-yl)methanesulfonamide hydrochloride] for: Treatment of chondrosarcoma
Date of first decision: 13/05/2011, Withdrawn,