Orphan designation: Atrasentan Treatment of primary IgA nephropathy, 10/12/2021 Positive
Annual report of the Pharmacovigilance Inspectors Working Group for 2023
Annual report of the Pharmacovigilance Inspectors Working Group for 2023
Orphan designation: humanised IgG1 monoclonal antibody against annexin-A1 Treatment of pancreatic cancer, 15/02/2023 Positive
Orphan designation: dry extract from birch bark (DER 5-10 : 1), extraction solvent n-heptane 95% (w/w) (birch bark extract) Treatment of epidermolysis bullosa, 23/02/2011 Positive
Orphan designation: idebenone Treatment of Friedreich's ataxia, 08/03/2004 Positive
Orphan designation: idebenone Treatment of Duchenne muscular dystrophy, 20/03/2007 Positive
Orphan designation: Cyclo-L-glycyl-L-2-allylproline Treatment of Angelman syndrome, 06/01/2021 Positive
Orphan designation: Cyclo-L-glycyl-L-2-allylproline Treatment of Phelan-McDermid syndrome, 06/01/2021 Positive
Orphan designation: Cyclo-L-glycyl-L-2-allylproline Treatment of Pitt-Hopkins syndrome, 06/01/2021 Positive