New fee regulation: webinar for human industry stakeholders, Online, European Medicines Agency, Amsterdam, the Netherlands, from 24 October 2024, 10:30 (CEST) to 24 October 2024, 11:30 (CEST)
New chair elected for EMA’s Orphan Medicinal Products Committee
Organisation chart: Human Medicines
Human Medicines
Orphan designation: N-[(1R)-1-[(S)-(2-Chloro-3-fluorophenyl)hydroxymethyl]butyl]-7-fluoro-2,3-dihydro-2-oxo-1H-indole-4-carboxamide Treatment of congenital alpha-1 antitrypsin deficiency, 25/07/2024 Positive
Orphan designation: N,N'-([Cyclohexylmethylene]di-4,1-phenylene)bis(2-[1-pyrrolidinyl]acetamide) Treatment of Creutzfeldt-Jakob disease, 25/07/2024 Positive
Orphan designation: emavusertib Treatment of primary large B-cell lymphoma of immune-privileged sites, 25/07/2024 Positive
Orphan designation: Sodium phenylacetate,Ursodoxicoltaurine Treatment of Wolfram syndrome, 25/07/2024 Positive
Orphan designation: Adeno-associated virus vector serotype rh.10 containing the human FXN gene Treatment of Friedreich's ataxia, 25/07/2024 Positive
Orphan designation: 4-[[[4-[5-Chloro-2-[[trans-4-[[(1R)-2-methoxy-1-methyl-ethyl]amino]cyclohexyl]amino]-4-pyridinyl]-2-thiazolyl]amino]methyl]tetrahydro-2H-pyran-4-carbonitrile Treatment of peripheral T-cell lymphoma, 25/07/2024 Positive