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
Final Minutes – HMA-EMA joint Big Data Steering Group teleconference - 22 March 2024
Orphan designation: Autologous CD3-positive T-cells expressing a chimeric antigen receptor against B cell maturation agent Treatment of AL amyloidosis, 19/02/2024 Positive
Orphan designation: Human IgG1 monoclonal antibody targeting amyloid transthyretin Treatment of ATTR amyloidosis, 19/02/2024 Positive