Human medicines European public assessment report (EPAR): Ojemda, tovorafenib, Date of authorisation: 20/04/2026, Status: Authorised
Orphan designation: 2-((2-fluoro-4-iodophenyl)amino)-N-(2-hydroxyethoxy)-1-methyl-1H-pyrrolo[2,3-b]pyridine-3-carboxamide Treatment of neurofibromatosis type 1, 22/05/2023 Positive
Orphan designation: encaleret Treatment of hypoparathyroidism, 15/10/2021 Positive
Orphan designation: eltanexor Treatment of myelodysplastic syndromes, 21/06/2022 Positive
Orphan designation: (Z)-3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-1,2,4-triazol-1-yl)-N'-(pyrazin-2-yl)acrylohydrazide Treatment of diffuse large B-cell lymphoma, 22/08/2014 Positive
Orphan designation: selinexor Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma, 19/11/2014 Positive
Orphan designation: selinexor Treatment of myelofibrosis, 11/10/2022 Positive
Orphan designation: selinexor Treatment of glioma, 15/10/2021 Positive
Orphan designation: (Z)-3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-1,2,4-triazol-1-yl)-N'-(pyrazin-2-yl)acrylohydrazide Treatment of acute myeloid leukaemia, 22/08/2014 Positive
Orphan designation: beta-lapachone Treatment of primary sclerosing cholangitis, 20/06/2025 Positive