Orphan designation: cyclo(arginyl-glycyl-aspartyl-D-tyrosyl-lysyl)-nizaracianine-1 Treatment of head and neck cancer, 22/06/2026 Positive

Human medicines European public assessment report (EPAR): Yuflyma, adalimumab, Date of authorisation: 11/02/2021, Revision: 21, Status: Authorised

Human medicines European public assessment report (EPAR): Bomyntra, denosumab, Date of authorisation: 17/07/2025, Revision: 2, Status: Authorised

Orphan designation: (S)-N-(2-Methoxy-5-((5-(trifluoromethyl)pyridin-2-yl)oxy)phenyl)-3-methyl-2-oxoimidazolidine-4-carboxamide Treatment of malignant mesothelioma, 22/06/2026 Positive

Orphan designation: guide RNA against the human HAO1 gene, mRNA encoding an engineered variant of the Cas12 nuclease Treatment of primary hyperoxaluria, 22/06/2026 Positive

Orphan designation: adeno-associated viral vector serotype 8 containing the human CASQ2 gene Treatment of catecholaminergic polymorphic ventricular tachycardia, 22/06/2026 Positive

Orphan designation: herpes simplex virus, type 1, containing the human NGF gene Treatment of neurotrophic keratitis, 22/06/2026 Positive

Orphan designation: modified mRNA encoding human cystic fibrosis transmembrane conductance regulator Treatment of cystic fibrosis, 23/06/2026 Positive

Orphan designation: rozanolixizumab Treatment of myelin oligodendrocyte glycoprotein antibody-associated disease, 28/07/2026 Positive

Orphan designation: 1-({(5S,7S,8R)-8-Fluoro-3-[5-(2-hydroxypropan-2-yl)pyrazin-2-yl]-7-methyl-2-oxo-1-oxa-3-azaspiro[4.5]decan-7-yl}methyl)-1H-1,3-benzimidazole-6-carbonitrile Treatment of TRPV4-related neuromuscular disease, 28/07/2026 Positive