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
EMEA-003071-PIP02-23
Human medicines European public assessment report (EPAR): Mynzepli, aflibercept, Date of authorisation: 18/08/2025, Revision: 1, Status: Authorised
Orphan designation: leuconostoc citreum, strain G511, Live Treatment of primary sclerosing cholangitis, 21/11/2025 Positive
Human medicines European public assessment report (EPAR): Myclausen, mycophenolate mofetil, Date of authorisation: 07/10/2010, Revision: 17, Status: Authorised