Orphanet: Search for an orphan drug
Search for an orphan drug
87 Result(s)
(5R,5aR,8aR,9S)-9-[[4,6-O-[(R)-Ethylidene]-beta-D-glucopyranosyl]-oxy]-5-(4-({[(2,2-dimethyl-1,3-dioxolan-4-yl)methoxy]carbonyl}oxy)-3,5-dimethoxyphenyl)-5,8,8a,9-tetrahydroisobenzofuro[5,6-f][1,3]benzodioxol-6(5aH)-one
(R)-6-(2-fluorophenyl)-N-(3-(2-((2 -methoxyethyl)amino)ethyl)phenyl)-5,6-dihydrobenzo[h]quinazolin-2-amine dihydrochloride
(S)-10-[(dimethylamino)methyl]-4-ethyl-9-hydroxy-4-O-[alpha-(2'', 4'', 5'', 7''- tetranitro-9''-fluorenylideneaminooxy)propionyl]-1H-pyrano[3', 4', 6', 7']indolizino[1,2- beta]-quinoline-3, 14-(4H, 12H)-dione, hydrochloride
1-((2S,4S)-2-((((S)-(4-bromophenoxy)(((S)-1-oxo-1-(((S)-pentan-2-yl)oxy)propan-2-yl)amino)phosphoryl)oxy)methyl)-1,3-dioxolan-4-yl)-2-oxo-1,2-dihydropyrimidin-4-aminium chloride
2-(2-phenylvinyl)-4-[4-methylpiperazin-1-yl)]-6-(5-methyl-2H-pyrazol-3-yl-amino)-pyrimidine L(+) tartrate salt
4-Amino-1-[5-O-[(2R,4S)-2-oxido-4-(4-pyridinyl)-1,3,2-dioxaphosphorinan-2-yl]-D-arabinofuranosyl]- 2(1H)-pyrimidinone
4-[3,5-bis(trimethylsilyl)benzamido] benzoic acid
5,10,15,20-tetrakis(2,6-difluoro-3-N-methylsulfamoylphenyl) bacteriochlorin
5-amino-1-(2-methyl-1H-benzo[d]imidazol-5-yl)-1H-pyrazol-4-yl 1H-indol-2-yl ketone mono[(S)-2-hydroxysuccinate]
6-thio-2'-deoxyguanosine
A genetically modified Salmonella comprising of methioninase
An autologous T-cell immunotherapy (ET140203)
Becatecarin
Bicistronic messenger ribonucleic acid (mRNA) encoding for ZF-DNMT and ZF-KRAB proteins, encapsulated in a lipid nanoparticle (LNP)
Bintrafusp Alfa
Brivanib alaninate
Devimistat
Diaspirin cross-linked haemoglobin
Doxorubicin Transdrug
Doxorubicin carbon/iron magnetically targeted microparticles
Doxorubicin hydrochloride (in heat-sensitive liposomes)
Ethiodized oil injection
Ezurpimtrostat
Fibroblast growth factor receptor (FGFR) antagonist
Fibroblast growth factor receptor (FGFR) inhibitor
Fimaporfin
Florilglutamic acid (18F)
Fosgemcitabine palabenamide
Galunisertib
IgG1 humanized antibody directed against Carcinoembryonic Antigen-related Cell Adhesion Molecule (CEACAM)1, 3 and 5
Infigratinib
Ivosidenib
Lenvatinib
Mefuparib
Mertansine functionalised gold nanoconjugate
Mifamurtide
N- 1',2-dihydroxy- 1,2'-binapthalen -4'-yl -4-methoxybenzenesulfonamide
N-(4-(4-amino-5-(3-fluoro-4-((4-methylpyrimidin-2-yl)oxy)phenyl)-7-methyl-7H-pyrrolo[2,3-d]pyrimidin-6-yl)phenyl)methacrylamide hydrochloride
N-(6-(2,6-dichloro-3,5-dimethoxyphenyl)-1H-pyrazolo[3,4-b]pyridin-3-yl)-4-(3,3-dimethylpiperazin-1-yl)benzamide hydrochloride
N-[4-(3-amino-1H-indazol-4 yl)phenyl]-N'-(2-fluoro-5-methylphenyl) urea
N-{2-[(6-{[(2,6-dichloro-3,5-dimethoxyphenyl)carbamoyl](methyl)amino}pyrimidin-4-yl)amino]-5-(4-ethylpiperazin-1-yl)phenyl}prop-2-enamide
NGR-human Tumour Necrosis Factor
Namodenoson
Nanvuranlat
Nemorubicin hydrochloride
Nolatrexed
Pegylated arginine deiminase
Peretinoin
Porfimer sodium (for use with photodynamic therapy)
Ramucirumab
Recombinant human IL12/15-PDL1B HSV-1 oncolytic virus Injection
Recombinant oncolytic virus M1-c6v1
Rencofilstat
Resminostat
Seocalcitol
Silmitasertib
Sorafenib tosylate
Sulfonated monophosphorylated mannose oligosaccharide
Tegavivint
Thymalfasin
Tirapazamine
Tislelizumab
Tivantinib
Tremelimumab
Vaccinia GM-CSF/TK-deactivated virus
Veliparib
a humanized immunoglobulin IgG1 antibody with a kappa light chain (LC) which has high selectivity and specificity for cancer-specific plectin
an autologous T-cell immunotherapy (ECT204)
sodium 2-hydroxylinoleate
zanidatamab