Orphan designation: antisense oligonucleotide targeting exon 73 in the COL7A1 gene Treatment of epidermolysis bullosa, 12/10/2017 Withdrawn
Orphan designation: Recombinant adeno-associated viral vector serotype S3 containing codon-optimised expression cassette encoding human coagulation factor IX variant Treatment of haemophilia B, 26/10/2018 Withdrawn
Human medicines European public assessment report (EPAR): Febuxostat Viatris (previously Febuxostat Mylan), febuxostat, Date of authorisation: 15/06/2017, Revision: 13, Status: Authorised
Orphan designation: Repagermanium Treatment of focal segmental glomerulosclerosis, 19/02/2024 Positive
Orphan designation: Andecaliximab Treatment of fibrodysplasia ossificans progressiva, 19/02/2024 Positive
Clinical Trials Information System (CTIS): Walk-in clinic - March 2024, Online, European Medicines Agency, Amsterdam, the Netherlands, Broadcast, from 12 March 2024, 16:00 (CET) to 12 March 2024, 17:00 (CET)
Orphan designation: Raludotatug deruxtecan Treatment of ovarian cancer, 19/02/2024 Positive
Orphan designation: Treatment of small cell lung cancer, 19/02/2024 Positive
Orphan designation: carboplatin Treatment of glioma, 19/02/2024 Positive
Orphan designation: Plerixafor Treatment of acute respiratory distress syndrome (ARDS), 19/02/2024 Positive