Orphan designation: Adeno-associated virus serotype ATC-0187 vector containing the human BAG3 gene Treatment of BAG3 dilated cardiomyopathy, 09/01/2026 Positive
Orphan designation: domatinostat Treatment of osteosarcoma, 09/01/2026 Positive
Orphan designation: autologous human T cells transduced with a lentiviral vector encoding a chimeric antigen receptor against the ERBB family Treatment of Fanconi anaemia, 09/01/2026 Positive
Orphan designation: spironolactone Treatment of central serous chorioretinopathy, 09/01/2026 Positive
Veterinary medicines European public assessment report (EPAR): Equidormin, midazolam, Status: Application withdrawn
Orphan designation: Phe-Thr-Thr-Phe-Thr-Val-Thr Treatment of idiopathic pulmonary fibrosis, 09/01/2026 Positive
Orphan designation: opamtistomig Treatment of extrapulmonary neuroendocrine carcinoma, 09/01/2026 Positive
Orphan designation: (4Z,7R,8E,10E,12Z,14S,16Z,19Z)-7,14-Dihydroxydocosa-4,8,10,12,16,19-hexaenoic acid Treatment of spinal cord injury, 23/02/2026 Positive
Human medicines European public assessment report (EPAR): Mycophenolate mofetil Teva, mycophenolate mofetil, Date of authorisation: 21/02/2008, Revision: 28, Status: Authorised
Orphan designation: spironolactone Treatment of limbal stem cell deficiency, 23/02/2026 Positive