Orphan designation: N-(2-Methoxyethyl)-6-methyl-N-[(3-methyl-2-thienyl)methyl]-2-oxo-1,2-dihydropyridine-4-carboxamide Treatment of glycogen storage disease type IV, 16/01/2025 Positive
Orphan designation: volixibat potassium Treatment of primary biliary cholangitis, 16/01/2025 Positive
Orphan designation: Allogenic umbilical cord-derived osteoblast cells Treatment of non-traumatic osteonecrosis, 16/01/2025 Positive
Orphan designation: Elraglusib Treatment of soft tissue sarcoma, 16/01/2025 Positive
Orphan designation: [4-(Methyl-1H-pyrazol-4-yl)-benzyl]-(6[7-(3-pyrrolidin-1-yl-propoxy)-imidazo[1,2-a]pyridin-3-yl]-pyrimidin-4-yl]-amine Treatment of gastrointestinal stromal tumours, 16/01/2025 Positive
Orphan designation: Elraglusib Treatment of pancreatic cancer, 16/01/2025 Positive
Orphan designation: Alvelestat Treatment of congenital alpha-1 antitrypsin deficiency, 16/01/2025 Positive
Orphan designation: Camostat mesilate Treatment of chronic pancreatitis, 16/01/2025 Positive
Data Analysis and Real World Interrogation Network (DARWIN EU)
DARWIN EU: Making health data count