Orphan designation: propagermanium Treatment of focal segmental glomerulosclerosis, 19/11/2018 Withdrawn

Orphan designation: 2'-O-4'-C-(S)-Ethyl-P-thioadenylyl-(3'-O->5'-O)-2'-O-4'-C-(S)-ethyl-P-thioguanylyl-(3'-O->5'-O)-2'-O-methyl-P-thiocytidylyl-(3'-O->5'-O)-2'-fluoro-P-thioadenylyl-(3'-O->5'-O)-2'-fluoro-P-thiocytidylyl-(3'-O->5'-O)-2'-fluoro-P-thiouridylyl-(3'-O->5'-O)-2'-O-methyl-P-thiouridylyl-(3'-O->5'-O)-2'-O-4'-C-(S)-ethyl-P-thiouridylyl-(3'-O->5'-O)-2'-O-4'-C-(S)-ethyl-adenosine Treatment of autosomal dominant polycystic kidney disease, 13/12/2025 Positive

Orphan designation: monepantel Treatment of amyotrophic lateral sclerosis, 13/12/2024 Positive

Orphan designation: allogeneic cardiosphere-derived cells Treatment of Duchenne muscular dystrophy, 13/12/2025 Positive

Orphan designation: diazoxide choline Treatment of glycogen storage disease type I, 13/12/2024 Positive

Information on the Member States requirement for the nomination of a pharmacovigilance (PhV) contact person at national level

Orphan designation: 4-(4-(2-(Diethylamino)ethoxy)phenyl)-1-(4-methoxybenzyl)-1H-1,2,3-triazol-5-amine Treatment of neurofibromatosis type 2, 13/12/2024 Positive

Orphan designation: divesiran Treatment of polycythaemia vera, 13/12/2024 Positive

Instrument for Pre-accession Assistance (IPA) EMA training on veterinary aspects of AMR and the One Health approach, European Medicines Agency, Amsterdam, the Netherlands, 21 October 2024

Instrument for Pre-accession Assistance (IPA) advanced EMA training on Clinical Trials, Pharmacovigilance and Shortages , European Medicines Agency, Amsterdam, the Netherlands, from 16 November 2023 to 17 November 2023