For help on how to get the results you want, see our search tips.
23 results
Categories
Human Remove Human filter
-
List item
Orphan designation: Garadacimab for: Treatment of hereditary angioedema
Date of designation: 10/12/2021, Positive, Last updated: 13/04/2022 -
List item
Orphan designation: Macitentan for: Treatment of chronic thromboembolic pulmonary hypertension
Date of designation: 10/12/2021, Positive, Last updated: 13/04/2022 -
List item
Orphan designation: Allogenic fetal mesenchymal stem cells for: Treatment of osteogenesis imperfecta
Date of designation: 10/12/2021, Positive, Last updated: 05/05/2022 -
List item
Orphan designation: Autologous T cells ex vivo modified with a lentiviral vector encoding a chimeric antigen receptor specific for CD1a for: Treatment of acute lymphoblastic leukaemia
Date of designation: 10/12/2021, Positive, Last updated: 13/04/2022 -
List item
Orphan designation: Vatiquinone for: Treatment of Alpers-Huttenlocher syndrome
Date of designation: 10/12/2021, Positive, Last updated: 13/04/2022 -
List item
Orphan designation: Retinol palmitate for: Prevention of bronchopulmonary dysplasia
Date of designation: 10/12/2021, Positive, Last updated: 13/04/2022 -
List item
Orphan designation: Anti-(endothelin-1 receptor subtype A) IgG4 humanised monoclonal antibody for: Treatment of pulmonary arterial hypertension
Date of designation: 10/12/2021, Positive, Last updated: 13/04/2022 -
List item
Orphan designation: 3-(ethoxydifluoromethyl)-6-(5-fluoro-6-(2,2,2-trifluoroethoxy)pyridin-3-yl)-[1,2,4]triazolo[4,3-a]pyrazine for: Treatment of SCN2A developmental and epileptic encephalopathy
Date of designation: 10/12/2021, Positive, Last updated: 13/04/2022 -
List item
Orphan designation: 3-(ethoxydifluoromethyl)-6-(5-fluoro-6-(2,2,2-trifluoroethoxy)pyridin-3-yl)-[1,2,4]triazolo[4,3-a]pyrazine for: Treatment of SCN8A developmental and epileptic encephalopathy
Date of designation: 10/12/2021, Positive, Last updated: 19/04/2022 -
List item
Orphan designation: Crofelemer for: Treatment of short bowel syndrome
Date of designation: 10/12/2021, Positive, Last updated: 19/04/2022 -
List item
Orphan designation: Atrasentan for: Treatment of primary IgA nephropathy
Date of designation: 10/12/2021, Positive, Last updated: 19/04/2022 -
List item
Orphan designation: copper (64Cu) chloride for: Treatment of glioma
Date of designation: 10/12/2021, Positive, Last updated: 20/04/2022 -
List item
Orphan designation: Ribonucleoprotein complex composed of two sgRNA and a Cas9 nuclease targeting the human COL7A1 gene for: Treatment of epidermolysis bullosa
Date of designation: 10/12/2021, Positive, Last updated: 20/04/2022 -
List item
Orphan designation: N-[1-((5-cyanopyridin-2-yl)methyl)-1H-pyrazol- 3-yl]-2-[4-(1-(trifluoromethyl)cyclopropyl) phenyl]acetamide for: Treatment of epileptic encephalopathy with continuous spike-and-wave during sleep
Date of designation: 10/12/2021, Positive, Last updated: 21/04/2022 -
List item
Orphan designation: 6-(4-(tert-butyl)phenoxy)pyridin-3-amine for: Treatment of acute lymphoblastic leukaemia
Date of designation: 10/12/2021, Positive, Last updated: 28/04/2022 -
List item
Orphan designation: Chimeric peptide of human glucagon-like peptide-1, glucagon and gastric inhibitory polypeptide analogues linked to a human immunoglobulin Fc fragment for: Treatment of primary sclerosing cholangitis
Date of designation: 10/12/2021, Positive, Last updated: 28/04/2022 -
List item
Orphan designation: Cedazuridine, decitabine for: Treatment of acute myeloid leukaemia
Date of designation: 10/12/2021, Positive, Last updated: 28/04/2022 -
List item
Orphan designation: Norrin (25-133), Lys86Pro for: Treatment of familial exudative vitreoretinopathy
Date of designation: 10/12/2021, Positive, Last updated: 03/05/2022 -
List item
Orphan designation: Unesbulin for: Treatment of soft tissue sarcoma
Date of designation: 10/12/2021, Positive, Last updated: 03/05/2022 -
List item
Orphan designation: Adeno-associated virus vector serotype 9 encoding the human GRN gene for: Treatment of frontotemporal dementia
Date of designation: 10/12/2021, Positive, Last updated: 03/05/2022 -
List item
Orphan designation: 2’-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3’-O->5’-O)-2’-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-(3’-O->5’-O)-2’-O-(2-methoxyethyl)-P-thioguanylyl-(3’-O->5’-O)-2’-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3’-O->5’-O)-2’-O-(2-methoxyethyl)-P-thioadenylyl-(3’-O->5’-O)-2’-O-(2-methoxyethyl)-P-thioadenylyl-(3’-O->5’-O)-2’-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3’-O->5’-O)-2’-O-(2-methoxyethyl)-P-thioadenylyl-(3’-O->5’-O)-2’-O-(2-methoxyethyl)-P-thioguanoylyl-(3’-O->5’-O)-2’-O-(2-methoxyethyl)-P-thioadenylyl-(3’-O->5’-O)-2’-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-(3’-O->5’-O)-2’-O-(2-methoxyethyl)-P-thioguanylyl-(3’-O->5’-O)-2’-O-(2-methoxyethyl)-P-thioguanylyl ... for: Treatment of cystic fibrosis
Date of designation: 10/12/2021, Positive, Last updated: 05/05/2022 -
List item
Orphan designation: Troriluzole hydrochloride for: Treatment of spinocerebellar ataxia (updated)
Date of designation: 10/12/2021, Positive, Last updated: 08/05/2023 -
List item
Orphan designation: Octreotide acetate for: Treatment of idiopathic intracranial hypertension
Date of designation: 10/12/2021, Positive, Last updated: 05/05/2022