Human medicines European public assessment report (EPAR): Lazcluze, lazertinib, Date of authorisation: 20/01/2025, Revision: 3, Status: Authorised
Orphan designation: puliretgene parvec Treatment of inherited retinal dystrophy due to dysfunction in the CYP4V2-gene, 12/09/2025 Positive
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