For help on how to get the results you want, see our search tips.
1969 results
Medicine
Orphan designations Remove Orphan designations filter
Shortages Remove Shortages filter
Periodic safety update report single assessments Remove Periodic safety update report single assessments filter
Direct healthcare professional communication Remove Direct healthcare professional communication filter
Withdrawn applications Remove Withdrawn applications filter
Categories
Human Remove Human filter
Orphan designation status
Positive Remove Positive filter
-
List item
Orphan designation: Dodecyl creatine ester, dodecyl creatine ester hydrochloride for: Treatment of creatine deficiency syndromes
Date of designation: 19/02/2021, Positive, Last updated: 06/02/2023 -
List item
Orphan designation: polyethylene glycol-modified human recombinant truncated cystathionine beta-synthase (pegtibatinase) for: Treatment of homocystinuria
Date of designation: 30/05/2016, Positive, Last updated: 06/02/2023 -
List item
Orphan designation: Volanesorsen sodium for: Treatment of familial partial lipodystrophy
Date of designation: 14/07/2016, Positive, Last updated: 02/02/2023 -
List item
Orphan designation: Phosphorothioate oligonucleotide targeted to apolipoprotein C-III (volanesorsen) for: Treatment of familial chylomicronaemia syndrome
Date of designation: 19/02/2014, Positive, Last updated: 02/02/2023 -
List item
Orphan designation: Allogeneic Epstein-Barr virus specific cytotoxic T lymphocytes (Tabelecleucel) for: Treatment of post-transplantation lymphoproliferative disorders
Date of designation: 21/03/2016, Positive, Last updated: 30/01/2023 -
List item
Orphan designation: Ex-vivo-expanded autologous keratinocytes transduced with retroviral vector containing the COL7A1 gene (prademagene zamikeracel) for: Treatment of epidermolysis bullosa
Date of designation: 27/02/2017, Positive, Last updated: 30/01/2023 -
List item
Orphan designation: Bardoxolone methyl for: Treatment of autosomal dominant polycystic kidney disease
Date of designation: 20/08/2021, Positive, Last updated: 30/01/2023 -
List item
Orphan designation: Omaveloxolone for: Treatment of Friedreich’s ataxia
Date of designation: 27/06/2018, Positive, Last updated: 30/01/2023 -
List item
Orphan designation: Bardoxolone methyl for: Treatment of Alport syndrome
Date of designation: 25/05/2018, Positive, Last updated: 30/01/2023 -
List item
Orphan designation: Rilonacept for: Treatment of idiopathic pericarditis
Date of designation: 06/01/2021, Positive, Last updated: 30/01/2023 -
List item
Orphan designation: propagermanium for: Treatment of focal segmental glomerulosclerosis
Date of designation: 19/11/2018, Positive, Last updated: 25/01/2023 -
List item
Orphan designation: Recombinant human mesencephalic astrocyte-derived neurotrophic factor for: Treatment of retinitis pigmentosa
Date of designation: 24/04/2015, Positive, Last updated: 24/01/2023 -
List item
Orphan designation: (S)-5-(1-(6-chloro-2-oxo-1,2-dihydroquinolin-3-yl)ethylamino-1-methyl-6-oxo-1,6-dihydropyridine-2-carbonitrile (olutasidenib) for: Treatment of acute myeloid leukaemia
Date of designation: 29/05/2019, Positive, Last updated: 23/01/2023 -
List item
Orphan designation: Ramiprilat for: Treatment of Stargardt's disease
Date of designation: 12/03/2013, Positive, Last updated: 17/01/2023 -
List item
Orphan designation: Herpes simplex virus 1 expressing the human CFTR gene for: Treatment of cystic fibrosis
Date of designation: 26/03/2021, Positive, Last updated: 17/01/2023 -
List item
Orphan designation: Replication-incompetent, non-integrating, herpes simplex virus 1 vector expressing the human transglutaminase-1 enzyme (Inetagugene geperpavec) for: Treatment of autosomal recessive congenital ichthyosis
Date of designation: 13/11/2019, Positive, Last updated: 13/01/2023 -
List item
Orphan designation: Pegylated adrenomedullin for: Treatment of acute respiratory distress syndrome (ARDS)
Date of designation: 27/07/2020, Positive, Last updated: 22/12/2022 -
List item
Orphan designation: Human allogeneic bone-marrow-derived osteoblastic-like cells for: Treatment of non-traumatic osteonecrosis
Date of designation: 05/08/2013, Positive, Last updated: 22/12/2022 -
List item
Orphan designation: Human allogeneic bone-marrow-derived osteoblastic cells for: Treatment of osteogenesis imperfecta
Date of designation: 10/08/2015, Positive, Last updated: 22/12/2022 -
List item
Orphan designation: bevacizumab for: Treatment of hereditary haemorrhagic telangiectasia
Date of designation: 16/12/2014, Positive, Last updated: 19/12/2022 -
List item
Orphan designation: 4-[131I] Iodo-L-phenylalanine for: Treatment of glioma
Date of designation: 11/04/2006, Positive, Last updated: 19/12/2022 -
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 diffuse large B-cell lymphoma
Date of designation: 16/12/2014, Positive, Last updated: 16/12/2022 -
List item
Orphan designation: pentosan polysulfate sodium for: Treatment of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)
Date of designation: 21/08/2020, Positive, Last updated: 15/12/2022 -
List item
Orphan designation: pentosan polysulfate sodium for: Treatment of mucopolysaccharidosis type I
Date of designation: 19/11/2014, Positive, Last updated: 15/12/2022 -
List item
Orphan designation: edaravone for: Treatment of amyotrophic lateral sclerosis
Date of designation: 16/12/2014, Positive, Last updated: 15/12/2022