Drug General Information
Drug ID
D0V1UW
Former ID
DAP000774
Drug Name
Nitisinone
Synonyms
Nitisinona; Nitisinonum; Nitisone; Orfadin; Nitisinone [INN]; SC 0735; FE-0200; Nitisinone (NTBC); Nitisinone [USAN:INN]; Orfadin (TN); Nitisinone (JAN/USAN/INN); 2-(2-Nitro-4-trifluoromethylbenzoyl)cyclohexane-1,3-dione; 2-(2-nitro-4-trifluoromethylbenzoyl)-1,3-cyclohexanedione; 2-(Alpha,alpha,alpha-trifluoro-2-nitro-p-tuluoyl)-1,3-cyclohexanedione; 2-[2-nitro-4-(trifluoromethyl)benzoyl]cyclohexane-1,3-dione; 2-{[2-nitro-4-(trifluoromethyl)phenyl]carbonyl}cyclohexane-1,3-dione
Drug Type
Small molecular drug
Indication Hereditary tyrosinemia type 1 [ICD9: 270.2; ICD10:E70.2] Approved [538562], [541915]
Therapeutic Class
Anticancer Agents
Structure
Download
2D MOL

3D MOL

Formula
C14H10F3NO5
InChI
InChI=1S/C14H10F3NO5/c15-14(16,17)7-4-5-8(9(6-7)18(22)23)13(21)12-10(19)2-1-3-11(12)20/h4-6,12H,1-3H2
InChIKey
OUBCNLGXQFSTLU-UHFFFAOYSA-N
CAS Number
CAS 104206-65-7
PubChem Compound ID
PubChem Substance ID
ChEBI ID
ChEBI:50378
SuperDrug ATC ID
A16AX04
SuperDrug CAS ID
cas=104206657
Target and Pathway
Target(s) 4-hydroxyphenylpyruvate dioxygenase Target Info Inhibitor [535880], [536692]
KEGG Pathway Ubiquinone and other terpenoid-quinone biosynthesis
Tyrosine metabolism
Phenylalanine metabolism
Metabolic pathways
References
Ref 538562FDA Approved Drug Products from FDA Official Website. 2009. Application Number: (NDA) 021232.
Ref 541915(http://www.guidetopharmacology.org/) Nucleic Acids Res. 2015 Oct 12. pii: gkv1037. The IUPHAR/BPS Guide to PHARMACOLOGY in 2016: towards curated quantitative interactions between 1300 protein targets and 6000 ligands. (Ligand id: 6834).
Ref 5358804-Hydroxyphenylpyruvate dioxygenase as a drug discovery target. Drug News Perspect. 2003 Oct;16(8):493-6.
Ref 536692Experience of nitisinone for the pharmacological treatment of hereditary tyrosinaemia type 1. Expert Opin Pharmacother. 2008 May;9(7):1229-36.

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