Orphan designation: curcumin Treatment of Charcot-Marie-Tooth disease, 18/07/2025 Positive

Orphan designation: ribavirin Treatment of hepatitis E, 18/07/2025 Positive

Orphan designation: avutometinib,defactinib Treatment of ovarian cancer, 18/07/2025 Positive

Orphan designation: palmitoyl-conjugated tricyclo-DNA antisense oligonucleotide 5'-Palm-C6-*GGA GAT GgC AGT TTC-3 Treatment of Duchenne muscular dystrophy, 18/07/2025 Positive

Orphan designation: luminol monosodium Treatment of Duchenne muscular dystrophy, 18/07/2025 Positive

Orphan designation: human bispecific monoclonal antibody targeting ALK1 and BMPRII Treatment of hereditary haemorrhagic telangiectasia, 18/07/2025 Positive

Orphan designation: adeno-associated viral vector serotype 9 containing the human CTNNB1 gene Treatment of autosomal dominant polycystic kidney disease, 18/07/2025 Positive

Orphan designation: octreotide hydrochloride Treatment of autosomal dominant polycystic kidney disease, 18/07/2025 Positive

Orphan designation: phosphorodiamidate morpholino oligonucleotide against the CUG repeat expansion of the DMPK gene mRNA transcript, conjugated to a cell penetrating peptide Treatment of dystrophic myotonia, 18/07/2025 Positive

Orphan designation: resecabtagene autoleucel Treatment of idiopathic inflammatory myopathy, 18/07/2025 Positive