Orphan designation: brepocitinib Treatment of idiopathic inflammatory myopathy, 22/08/2025 Positive
Orphan designation: antisense oligonucleotide against ABCA4 pre-mRNA Treatment of non-syndromic inherited retinal dystrophies due to defects in the ABCA4 gene, 21/11/2025 Positive
Orphan designation: zanidatamab Treatment of oesophageal cancer, 21/11/2025 Positive
Orphan designation: temozolomide Treatment of glioma, 22/08/2025 Positive
Orphan designation: varegacestat Treatment of soft tissue sarcoma, 22/08/2025 Positive
Orphan designation: zilurgisertib Treatment of fibrodysplasia ossificans progressiva, 22/08/2025 Positive
Orphan designation: sonpiretigene isteparvovec Treatment of syndromic inherited retinal dystrophies of the cone-dominant phenotype, 22/08/2025 Positive
Orphan designation: sonpiretigene isteparvovec Treatment of non-syndromic inherited retinal dystrophies of the rod-dominant phenotype, 22/08/2025 Positive
Orphan designation: methotrexate Treatment of primary large B-cell lymphoma of immune-privileged sites, 22/08/2025 Positive
Orphan designation: sonpiretigene isteparvovec Treatment of non-syndromic inherited retinal dystrophies of the cone-dominant phenotype, 22/08/2025 Positive