Orphan designation: methotrexate Treatment of non-syndromic inherited retinal dystrophies of the rod-dominant phenotype, 18/07/2025 Positive
Orphan designation: sonlicromanol hydrochloride Treatment of inherited mitochondrial oxidative phosphorylation defects, 18/07/2025 Positive
Orphan designation: Ivosidenib Treatment of myelodysplastic syndromes, 18/07/2025 Positive
Orphan designation: telitacicept Treatment of myasthenia gravis, 18/07/2025 Positive
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