IRIS guide for applicants - How to create and submit scientific applications, for industry and individual applicants
Early dialogue with healthcare professional organisations for marketing authorisation applications: 1-year report
Opinion/decision on a Paediatric investigation plan (PIP): Uplizna, Inebilizumab, decision type: PM: decision on the application for modification of an agreed PIP, therapeutic area: Neurology, PIP number: P/0513/2023
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
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