Orphan designation: lanthanum carbonate octahydrate Treatment of primary hyperoxaluria, 09/01/2026 Positive
Orphan designation: sirolimus Treatment of osteosarcoma, 09/01/2026 Positive
EMEA-001811-PIP03-20
Human medicines European public assessment report (EPAR): Mysimba, naltrexone,bupropion, Date of authorisation: 26/03/2015, Revision: 32, Status: Authorised
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Veterinary medicines European public assessment report (EPAR): Equidormin, midazolam, Status: Application withdrawn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