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