Orphan designation: anti-SIGLEC8 IgG1 humanised monoclonal antibody Treatment of eosinophilic gastroenteritis, 19/02/2021 Withdrawn

Orphan designation: sabatolimab Treatment of myelodysplastic syndromes, 20/08/2021 Withdrawn

Orphan designation: Ilginatinib maleate Treatment of myelofibrosis, 25/07/2023 Withdrawn

Orphan designation: adeno-associated viral vector serotype 8 containing the human acid alpha-glucosidase gene Treatment of glycogen storage disease type II (Pompe's disease), 16/04/2018 Withdrawn

Orphan designation: navitoclax Treatment of myelofibrosis, 16/12/2019 Withdrawn

Orphan designation: acalabrutinib Treatment of mantle cell lymphoma, 21/03/2016 Withdrawn

Orphan designation: pegylated adrenomedullin Treatment of acute respiratory distress syndrome (ARDS), 27/07/2020 Withdrawn

Network Portfolio Roadmap

Human medicines European public assessment report (EPAR): Ucedane, carglumic acid, Date of authorisation: 23/06/2017, Revision: 14, Status: Authorised

Questions and answers on periodic safety update report single-assessment (PSUSA): Guidance document for assessors