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

Orphan designation: Humanised IgG1 kappa monoclonal antibody against erythropoietin isoform EV-3 Treatment of glioma, 23/02/2026 Positive

Orphan designation: N-[(1S)-1-(4-Tert-butylphenyl) ethyl]-2-(6,7-difluoro-1H-benzimidazol-1-yl)acetamide Treatment of erythromelalgia, 23/02/2026 Positive

Orphan designation: autologous peripheral blood-derived CD34+ haematopoietic stem and progenitor cells transduced with a lentiviral vector containing the murine Glb1 gene Treatment of Glb1-related disorders, 22/10/2025 Positive

Orphan designation: (S)-1-(2,2-Difluoroethyl)-3-(difluoromethyl)-N-(1-(3-(2-methoxypyridin-4-yl)-1,2,4-oxadiazol-5-yl)ethyl)-1H-pyrazole-5-carboxamide Treatment of KCNT1-related epilepsy and neurodevelopmental disorders, 21/11/2025 Positive

Orphan designation: vopimetostat Treatment of pancreatic cancer, 21/11/2025 Positive

Meeting highlights from the Pharmacovigilance Risk Assessment Committee (PRAC) 7-10 April 2026