Orphan designation: messenger ribonucleic acid coding for coiled-coil domain-containing protein 40 Treatment of primary ciliary dyskinesia, 25/07/2023 Positive
Concept paper for the development of a reflection paper on a tailored clinical approach in biosimilar development
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