For help on how to get the results you want, see our search tips.
1858 results
Categories
Human Remove Human filter
Medicine
Shortages Remove Shortages filter
Opinions on medicines for use outside EU Remove Opinions on medicines for use outside EU filter
Orphan designations Remove Orphan designations filter
Orphan designation status
Positive Remove Positive filter
-
List item
Orphan designation: Rezafungin acetate for: Treatment of invasive candidiasis (updated)
Date of designation: 06/01/2021, Positive, Last updated: 30/06/2022 -
List item
Orphan designation: Recombinant human monoclonal antibody to insulin receptor for: Treatment of congenital hyperinsulinism (updated)
Date of designation: 14/07/2016, Positive, Last updated: 30/06/2022 -
List item
Orphan designation: 2-methyl-1-[(4-[6-(trifluoromethyl)pyridin-2-yl]-6-{[2-(trifluoromethyl)pyridin-4-yl]amino}-1,3,5-triazin-2-yl)amino]propan-2-ol methanesulfonate (enasidenib) for: Treatment of acute myeloid leukaemia (updated)
Date of designation: 28/04/2016, Positive, Last updated: 29/06/2022 -
List item
Orphan designation: Mosunetuzumab for: Treatment of follicular lymphoma (updated)
Date of designation: 12/11/2021, Positive, Last updated: 23/06/2022 -
List item
Orphan designation: Protein-based delivery vector carrying a DNA payload encoding an RNA-guided nuclease that targets stx genes of Shiga toxin-producing Escherichia coli for: Prevention of haemolytic uraemic syndrome (updated)
Date of designation: 21/08/2020, Positive, Last updated: 22/06/2022 -
List item
Orphan designation: Modified messenger ribonucleic acid encoding human propionyl-coenzyme A carboxylase alpha and beta subunits encapsulated into lipid nanoparticle for: Treatment of propionic acidaemia (updated)
Date of designation: 24/04/2019, Positive, Last updated: 22/06/2022 -
List item
Orphan designation: Modified mRNA encoding human methylmalonyl-coenzyme A mutase encapsulated into lipid nanoparticles for: Treatment of methylmalonic acidaemia (updated)
Date of designation: 25/05/2018, Positive, Last updated: 22/06/2022 -
List item
Orphan designation: Synthetic signal peptide of human mucin-1 (amino acids 1-21) for: Treatment of plasma cell myeloma (updated)
Date of designation: 15/01/2015, Positive, Last updated: 22/06/2022 -
List item
Orphan designation: Human autologous mesenchymal adult stem cells extracted from adipose tissue for: Treatment of anal fistula (updated)
Date of designation: 26/08/2005, Positive, Last updated: 14/06/2022 -
List item
Orphan designation: Adeno-associated viral vector serotype rh10 containing the human cholesterol 24-hydroxylase gene for: Treatment of Huntington’s disease (updated)
Date of designation: 01/04/2019, Positive, Last updated: 14/06/2022 -
List item
Orphan designation: Adeno-associated viral vector serotype 8 containing the human acid alpha-glucosidase gene for: Treatment of glycogen storage disease type II (Pompe's disease) (updated)
Date of designation: 16/04/2018, Positive, Last updated: 14/06/2022 -
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 (updated)
Date of designation: 28/02/2020, Positive, Last updated: 13/06/2022 -
List item
Orphan designation: Mecasermin rinfabate for: Prevention of retinopathy of prematurity in neonates of less than 32 weeks of gestational age (updated)
Date of designation: 28/08/2006, Positive, Last updated: 08/06/2022 -
List item
Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding galactosidase alpha for: Treatment of Fabry disease (updated)
Date of designation: 19/10/2020, Positive, Last updated: 08/06/2022 -
List item
Orphan designation: Autologous CD34+ cells transfected with a lentiviral vector containing codon optimised RPS19 gene for: Treatment of Diamond-Blackfan anaemia (updated)
Date of designation: 20/08/2021, Positive, Last updated: 08/06/2022 -
List item
Orphan designation: polatuzumab vedotin for: Treatment of diffuse large B-cell lymphoma (updated)
Date of designation: 16/04/2018, Positive, Last updated: 08/06/2022 -
List item
Orphan designation: Allogeneic retinal pigment epithelial cells genetically modified with a non-viral vector to express human alpha-L-iduronidase for: Treatment of mucopolysaccharidosis type I
Date of designation: 15/10/2021, Positive, Last updated: 24/05/2022 -
List item
Orphan designation: Allogeneic retinal pigment epithelial cells genetically modified with a non-viral vector to express beta-domain deleted human factor VIII for: Treatment of haemophilia A
Date of designation: 09/12/2020, Positive, Last updated: 24/05/2022 -
List item
Orphan designation: Tipifarnib for: Treatment of peripheral T-cell lymphoma
Date of designation: 19/10/2020, Positive, Last updated: 24/05/2022 -
List item
Orphan designation: 6-((3S,4S)-4-methyl-1-(pyrimidin-2-yl-methyl)pyrrolidin-3-yl)-3-tetrahydropyran-4-yl-7H-imidazo(1,5-a)pyrazin-8-one for: Treatment of sickle cell disease
Date of designation: 21/08/2020, Positive, Last updated: 24/05/2022 -
List item
Orphan designation: 3-(3-(3,5-bis(trifluoromethyl)phenyl)-1h-pyrazol-1-yl)propanoic acid for: Treatment of Stargardt's disease
Date of designation: 24/04/2019, Positive, Last updated: 24/05/2022 -
List item
Orphan designation: 6-(R)-methyl-5-O-(5-amino-5,6-dideoxy-alpha-L-talofuranosyl)-paromamine sulfate for: Treatment of cystic fibrosis
Date of designation: 26/10/2018, Positive, Last updated: 24/05/2022 -
List item
Orphan designation: 6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-α-L-talofuranosyl)-paromamine sulfate for: Treatment of mucopolysaccharidosis type I
Date of designation: 22/09/2016, Positive, Last updated: 24/05/2022 -
List item
Orphan designation: N-methyl-4-({4-[({3-methyl(methylsulfonyl)aminopyrazin-2-yl}methyl)amino]-5-(trifluoromethyl)pyrimidin-2-yl}amino)benzamide hydrochloride (defactinib) for: Treatment of ovarian cancer
Date of designation: 15/01/2015, Positive, Last updated: 24/05/2022 -
List item
Orphan designation: Tipifarnib for: Treatment of acute myeloid leukaemia
Date of designation: 10/03/2005, Positive, Last updated: 24/05/2022