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Orphan designations
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Last updated 11 days ago
Orphan designation: infliximab Treatment of Kawasaki disease, 20/05/2026 Positive
11 days ago
Orphan designation: infliximab Treatment of Kawasaki disease, 20/05/2026 Positive
Orphan designation: allogeneic human induced pluripotent stem cell-derived photoreceptor precursor cells Treatment of non-syndromic inherited retinal dystrophies of the rod-dominant phenotype, 20/05/2026 Positive
11 days ago
Orphan designation: allogeneic human induced pluripotent stem cell-derived photoreceptor precursor cells Treatment...
Orphan designation: humanised IgG1 monoclonal antibody against delta-like ligand 3, octakis(thioether) with N-[6-(2-mercapto-5-pyrimidinyl)-1-oxo-5-hexyn-1-yl]-L-valyl-N6,N6-dipropyl-L-lysyl-N-[[3-[(7S)-7-ethyl-7,8,11,13-tetrahydro-7-hydroxy-8,11-dioxo-10
11 days ago
Orphan designation: humanised IgG1 monoclonal antibody against delta-like ligand 3, octakis(thioether) with...
Orphan designation: allogeneic human Wharton's jelly-derived mesenchymal stem cells Treatment of Charcot-Marie-Tooth disease, 20/05/2026 Positive
11 days ago
Orphan designation: allogeneic human Wharton's jelly-derived mesenchymal stem cells Treatment of Charcot-Marie-Tooth...
Orphan designation: peptide-oligonucleotide conjugate against CNOT3 mRNA Treatment of inherited retinal dystrophies due to mutation in the PRPF31 gene, 20/05/2026 Positive
11 days ago
Orphan designation: peptide-oligonucleotide conjugate against CNOT3 mRNA Treatment of inherited retinal dystrophies...
Orphan designation: allogeneic peripheral blood mononuclear cells incubated in vitro with mitomycin C Treatment of in solid organ transplantation, 20/05/2026 Positive
11 days ago
Orphan designation: allogeneic peripheral blood mononuclear cells incubated in vitro with mitomycin...
Orphan designation: copper metabolism disorder Treatment of Wilson's disease, 20/05/2026 Positive
20 days ago
Orphan designation: copper metabolism disorder Treatment of Wilson's disease, 20/05/2026 Positive
Orphan designation: quemliclustat Treatment of pancreatic cancer, 20/05/2026 Positive
20 days ago
Orphan designation: quemliclustat Treatment of pancreatic cancer, 20/05/2026 Positive
Orphan designation: serabelisib Treatment of PIK3CA related overgrowth spectrum, 20/05/2026 Positive
20 days ago
Orphan designation: serabelisib Treatment of PIK3CA related overgrowth spectrum, 20/05/2026 Positive
Orphan designation: alixorexton Treatment of narcolepsy, 20/05/2026 Positive
20 days ago
Orphan designation: alixorexton Treatment of narcolepsy, 20/05/2026 Positive
Orphan designation: allogeneic human induced pluripotent stem cell-derived photoreceptor precursor cells Treatment of syndromic inherited retinal dystrophies of the rod-dominant phenotype, 20/05/2026 Positive
20 days ago
Orphan designation: allogeneic human induced pluripotent stem cell-derived photoreceptor precursor cells Treatment...
Orphan designation: antisense oligonucleotide against USH2A pre-mRNA Treatment of inherited retinal disease due to dysfunction in the USH2A gene, 20/05/2026 Positive
20 days ago
Orphan designation: antisense oligonucleotide against USH2A pre-mRNA Treatment of inherited retinal disease...