Orphan designation: Nomacopan Treatment in haematopoietic stem cell transplantation, 25/07/2023 Positive
Orphan designation: bezuclastinib Treatment of gastrointestinal stromal tumours, 20/03/2023 Positive
Orphan designation: rhizobium rhizogenes,lipopolysaccharide Treatment of congenital diaphragmatic hernia, 25/07/2023 Positive
Orphan designation: vonafexor Treatment of Alport syndrome, 25/07/2023 Positive
Orphan designation: Certepetide Treatment of pancreatic cancer, 13/10/2023 Positive
Orphan designation: taldefgrobep alfa Treatment of spinal muscular atrophy, 25/07/2023 Positive
Orphan designation: Indole-3-carboxaldehyde Treatment of primary CTLA-4 checkpoint related immunodeficiencies, 20/03/2023 Positive
Orphan designation: sildenafil citrate Treatment of Leigh syndrome, 13/10/2023 Positive
Orphan designation: Allogeneic peripheral blood-derived haematopoietic stem and progenitor cells, regulatory T cells and conventional T cells Treatment in haematopoietic stem cell transplantation, 25/07/2023 Positive
Orphan designation: Omadacycline Treatment of nontuberculous mycobacterial lung disease, 25/07/2024 Positive