Orphanet: Search for an orphan drug
Search for an orphan drug
254 Result(s)
(R)-1-(2,2-difluorobenzo [d][1,3] dioxol-5-yl)-N-(1-(2,3-dihydroxypropyl)-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl) cyclopropanecarboxamide (R)-2-(5-cyano-2-(6-(methoxycarbonyl)-7-methyl-3-oxo-8-(3-(trifluoromethyl)phenyl)-2,3,5,8-tetrahydro-[1,2,4]triazolo[4,3-a]pyrimidine-5-yl)phenyl)-N,N,N-trimethylethanaminium methanesulfonate dehydrate (S)-(-)-3-(4-aminophenyl)-2-methoxypropanoic acid (S)-8-{2-Amino-6-[1-(5-chloro-biphenyl-2-yl)-(R)-2,2,2-trifluoro-ethoxy]-pyrimidin-4-yl}-2,8-diaza-spiro[4.5]decane-3-carboxylic acid ethyl ester 11-(4-Dimethylamino-3-hydroxy-6-methyl-tetrahydro-pyran-2-yloxy)-2-ethyl-3,4,10-trihydroxy-3,5,6,8,10,12,14-heptamethyl-1-oxa-6-aza-cyclopentadecane-13,15-dione 2-((2-ethyl-6-(4-(2-(3-hydroxyazetidin-1-yl)-2-oxoethyl)-piperazin-1-yl)-8-methylimidazo[1,2-alpha]pyridin-3-yl)-(methyl)amino)-4-(4-fluorophenyl)-thiazole-5-carbonitrile 2-(2-chlorophenyl)-4-[3-(dimethylamino)phenyl]-5-methyl-1H-pyrazolo[4,3-c]pyridine-3,6(2H,5H)-dione 2-(7-ethoxy-4-(3-fluorophenyl)-1-oxophthalazin-2(1H)-yl)-N-methyl-N-(2-methylbenzo[d]oxazol-6-yl) acetamide 2-{[(1r,4r)-4-(((4-chlorophenyl)(phenyl)carbamoyloxy)methyl)cyclohexy]methoxy} acetic acid 3-(6-(1-(2,2-difluorobenzo [d] [1,3] dioxol-5-yl)cyclopropanecarboxamido)-3- methylpyridin-2-yl)benzoic acid 3-Pentylbenzeneacetic acid sodium salt 3-[4-(1H-imidazol-1-ylmethyl)phenyl]-5-(2-methylpropyl)thiophene-2-[(N-butyloxylcarbamate)-sulphonamide] sodium salt 4,6,4'-trymethylangelicin 4-[[(1S,4S)-5-[[4-[4-(oxazol-2-yl)phenoxy]phenyl]methyl]-2,5-diazabicyclo[2.2.1]hept-2-yl]methyl]benzoic acid 6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-alpha-L-talofuranosyl)-paromamine sulfate 8-cyclopentyl-1,3-dipropylxanthine Acetylcysteine Acetylleucine Adalimumab Alginate oligosaccharide (G-block) fragment Alglucerase Alpha-1 antitrypsin (inhalation use) Alpha-1 proteinase inhibitor (inhalation use) Ambrisentan Amikacin sulfate Amikacin sulfate (liposomal) Amiloride hydrochloride dihydrate Antagonist of the complement 5a receptor Arimoclomol citrate Ataluren Autologous CD34+ cells transduced with retroviral vector containing the human gp91 (phox) gene Autologous haematopoietic cells genetically modified with a lentiviral vector containing the human gp91(phox) gene Avacopan Avian polyclonal IgY antibody against Pseudomonas aeruginosa Aviptadil Aztreonam lysinate (inhalation use) Bacterial lipase Beraprost sodium Beraprost sodium (modified release tablet) Bosentan Budesonide (oral use) Canakinumab Carbamic acid, [[4-[[3-[[4-[1-(4-hydroxyphenyl)-1-methylethyl] phenoxy]methyl]phenyl]methoxy]-phenyl]iminomethyl]-,ethyl ester Ciprofloxacin (inhalation use) Ciprofloxacin (liposomal) Colistimethate sodium Cyclo[L-alanyl-L-seryl-L-isoleucyl-L-prolyl-L-prolyl-L-glutaminyl-L-lysyl-L-tyrosyl-D-prolyl-L-prolyl-(2S)-2-aminodecanoyl-L-alpha-glutamyl-L-threonyl] acetate salt Cysteamine Cystic fibrosis transmembrane conductance regulator (CFTR) amplifier Denufosol tetrasodium Dexamethasone sodium phosphate encapsulated in human erythrocytes Dipalmitoylphosphatidylcholine, 1-palmitoyl-2-oleoyl-sn-glycero-3-phosphoglycerol, sodium salt, synthetic surfactant protein C analogue and synthetic surfactant protein B analogue Dornase alfa Drotrecogin alfa (activated) Duramycin Elafin Elexacaftor/Tezacaftor/Ivacaftor Eliglustat Eluforsen Engineered protein inhibitor of human neutrophil elastase Epoprostenol Etanercept Fixed-dose combination of fosfomycin disodium and tobramycin Gallium citrate Givinostat Glutathione Granulocyte-macrophage colony-stimulating factor Gusperimus trihydrochloride Heparin sodium Humanised anti-alpha v beta 6 monoclonal antibody Hydroxy-Propyl-Beta-Cyclodextrin Hypothiocyanite / lactoferrin Iloprost Iloprost inhalation solution Iloprost trometamol Imatinib mesilate Imiglucerase Interferon beta Interferon gamma Interferon gamma 1a Interferon gamma 1b Ivacaftor L-Lysine-N-acetyl-L-cysteinate L-Pyr-L-Glu-L-Gln-L-Leu-L-Glu-L-Arg-L-Ala-L-Leu-L-Asn-L-Ser-L-Ser Lenabasum Liprotamase Lisuride hydrogen maleate Lucinactant Lumacaftor / Ivacaftor Lusupultide Macitentan Mannitolum Meloxicam Mepolizumab Methyl 4,6-diamino-2-[1-(2-fluorobenzyl)-1H-pyrazolo[3,4-b]pyridine-3-yl]-5-pyrimidinyl(methyl)carbamate Miglustat Molgramostim Multilamellar microvesicle comprising phosphatidylcholine, sphingomyelin, phosphatidylethanolamine, phosphatidylserine, phosphatidylinositol and cholesterol N-(tert-butylcarbamoyl)-5-cyano-2-((4'-(difluoromethoxy)-[1,1'-biphenyl]-3-yl)oxy)benzenesulfonamide Nafamostat mesilate Nintedanib Nitric oxide Octovalent Pseudomonas aeruginosa O-polysaccharie-toxin A conjugate vaccine Pegylated recombinant anti-Pseudomonas aeruginosa PcrV Fab' antibody Phosphoinositide 3-kinase gamma peptide Pirfenidone Plasmid DNA encoding the human cystic fibrosis transmembrane conductance regulator gene complexed with a non-viral, cationic lipid based gene transfer agent Platelet-derived growth factor (PDGF) receptor kinase inhibitor Pulmonary surfactant replacement, porcine Ralinepag Recombinant dog gastric lipase Recombinant human CXCL8 mutant Recombinant human acid ceramidase Recombinant human acid sphingomyelinase Recombinant human bile salt-stimulated lipase Recombinant human club cell 10 KDa protein (rhCC10) Recombinant human heat shock protein 70 Recombinant human monoclonal antibody against mannan-binding lectin-associated serine protease-2 Recombinant human monoclonal antibody against transforming growth factor beta-1, 2 and 3 Recombinant human pentraxin-2 Recombinant microbial lipase Riociguat Rituximab Selexipag Sildenafil citrate Sinapultide, dipalmitoylphosphatidylcholine, palmitoyloleoylphosphatidylglycerol and palmitic acid Sirolimus Sitaxentan sodium Sodium cromoglicate Sodium nitrite Surface active extract of saline lavage of bovine lungs T-Cell Depleted Stem Cell Enriched Cellular Product From Peripheal Blood Stem Cells Tacrolimus Tamoxifen citrate Tanzisertib Teicoplanin Temocillin sodium Terguride Tezacaftor Tezacaftor / Ivacaftor Tilorone Tipelukast Tobramycin (inhalation powder) Tobramycin (inhalation solution) Tobramycin (inhalation use) Tobramycin (liposomal) Tocilizumab Tralokinumab Treprostinil diethanolamine (oral use) Treprostinil sodium (inhalation use) Ubenimex Ursodeoxycholic acid Vasoactive intestinal peptide (VIP)-elastin-like peptide (ELP) fusion protein adeno-associated viral vector serotype 9 containing the human glucocerebrosidase gene codon-optimised human cystic fibrosis transmembrane conductance regulator messenger ribonucleic acid complexed with lipid-based nanoparticles imlifidase ivacaftor, potassium(benzenesulfonyl)({[6-(3-{2-[1-(trifluoromethyl) cyclopropyl]ethoxy}-1H-pyrazol-1-yl)-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridin-3-yl]carbonyl})azanide, tezacaftor levofloxacin hemihydrate poly-N (acetyl, arginyl) glucosamine (PAAG) sodium nitrite and EDTA
