Orphan designation: zimberelimab Treatment of oesophageal cancer, 12/09/2025 Positive

Orphan designation: autologous peripheral blood-derived CD34+ haematopoietic stem and progenitor cells transduced with a lentiviral vector containing the human MAN2B1 gene Treatment of alpha-mannosidosis, 12/09/2025 Positive

Orphan designation: cyclo(arginyl-glycyl-aspartyl-D-tyrosyl-lysyl)-nizaracianine-1 Diagnosis of pancreatic cancer, 12/09/2025 Positive

Human medicines European public assessment report (EPAR): Axumin, fluciclovine (18F), Date of authorisation: 21/05/2017, Revision: 20, Status: Authorised

Orphan designation: domvanalimab Treatment of oesophageal cancer, 12/09/2025 Positive

Orphan designation: humanised IgG1 kappa monoclonal antibody against pregnancy-associated plasma protein A Treatment of autosomal dominant polycystic kidney disease, 12/09/2025 Positive

Orphan designation: biotin Treatment of biotin-thiamine-responsive basal ganglia disease (BTBGD), 12/09/2025 Positive

Orphan designation: human IgG1 monoclonal antibody against tumor necrosis factor receptor superfamily member 1B Treatment of cutaneous T-cell lymphoma, 12/09/2025 Positive

Orphan designation: zimberelimab Treatment of gastric cancer, 12/09/2025 Positive

Orphan designation: adeno-associated virus vector serotype 6.2 containing human TERT gene Treatment of idiopathic pulmonary fibrosis, 12/09/2025 Positive