Orphan designation: motixafortide Treatment of patients undergoing haematopoietic stem cell transplantation, 12/01/2024 Positive
Orphan designation: mitapivat sulfate Treatment of thalassaemia intermedia and major, 12/01/2024 Positive
Orphan designation: Thiophene methylimidazole pentahydrogen Diagnosis of glioma, 12/01/2024 Positive
Orphan designation: Ersodetug Treatment of insulinoma, 12/01/2024 Positive
Orphan designation: Methyl-(1-{[6-{[(1S)-1-cyclopropylethyl]amino}-2-(pyrazolo[5,1-b][1,3]thiazol-7-yl)-pyrimidin-4-yl]carbonyl}piperidin-4-yl)carbamate mono(4-methylbenzenesulfonate) Treatment of eosinophilic granulomatosis with polyangiitis, 12/01/2024 Positive
Orphan designation: golcadomide hydrochloride Treatment of diffuse large B-cell lymphoma, 12/01/2024 Positive
Orphan designation: Human coagulation factor X Treatment of acquired factor X deficiency, 12/01/2024 Positive
Orphan designation: (4R)-3-(4-fluoro-2-hydroxyphenyl)-4-methyl-4,5-dihydro-1H-pyrazole-1-carboximidamide hydrochloride Treatment of systemic sclerosis, 12/01/2024 Positive
Orphan designation: mRNA encoding the human CFTR gene Treatment of cystic fibrosis, 19/02/2024 Positive
Human medicines European public assessment report (EPAR): Ambrisentan Mylan, ambrisentan, Date of authorisation: 20/06/2019, Revision: 5, Status: Authorised