Обновленный список препаратов, получивших статус орфанных
В октябре 2014 по данным Eurordis
Treatment of inhalation anthrax disease
Raxibacumab
Treatment of X-linked hypophosphataemia
Recombinant human monoclonal IgG1 antibody for fibroblast growth factor 23
Treatment of tenosynovial giant cell tumour, localised and diffused type
Recombinant human monoclonal antibody of the IgG1 kappa class against human macrophage colony-stimulating factor
Treatment of mucopolysaccharidosis type I
Recombinant human insulin receptor monoclonal antibody-fused-α-Liduronidase
Treatment of glioma
Recombinant human bone morphogenetic protein 4
Treatment of pyridoxamine 5'-phosphate oxidase deficiency
Pyridoxal 5'-phosphate
Treatment of short bowel syndrome
Oxalobacter formigenes strain HC-1
Treatment of Cushing's syndrome
Osilodrostat
Treatment of cystic fibrosis
Nitric oxide
Treatment of pancreatic cancer
Immunoglobulin G1, anti-(human tumour-associated calcium signaltransducer 2)(human-Mus musculus monoclonal hRS7 heavy chain), disulfide with human-Musmusculus monoclonal hRS7 k-chain, dimer, hexakis(thioether) with (4S)-4-[[[[4-[[(2S)-2-(4-aminobutyl)-2-[[2-[2-[[26-[4-[[[[4-[(3-mercapto-2,5-dioxo-1-pyrrolidinyl)methyl]cyclohexyl]carbonyl]amino]methyl]-1H-1,2,3-triazol-1-yl]-3,6,9,12,15,18,21,24-octaoxahexacos-1-yl]amino]-2-oxoethoxy]acetyl]amino]-1-oxoethyl]amino]phenyl]methoxy]carbonyl]oxy]-4,11-diethyl-9-hydroxy-1Hpyrano[3',4':6,7]indolizino[1,2-b]quinoline-3,14(4H,12H)-dione
Treatment of congenital hyperinsulinism
Glucagon
Treatment of cystinosis
Cysteamine hydrochloride
Treatment of limbal stem cell deficiency
Cultured allogeneic corneal limbal stem cells
Treatment of Dravet syndrome
Cannabidiol
Treatment of Crigler-Najjar syndrome
Adeno-associated viral vector serotype 8 containing the human UGT1A1 gene
Treatment of fragile X syndrome
Acamprosate calcium
Treatment of Leigh syndrome
(S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3-yl)chroman-2-carboxamide hydrochloride
Treatment of pemphigus
(S)-2-(1-((6-amino-5-cyanopyrimidin-4-yl)amino)ethyl)-4-oxo-3-phenyl-3,4-dihydropyrrolo[2,1-f][1,2,4]triazine-5-carbonitrile