nitisinone   Click here for help

GtoPdb Ligand ID: 6834

Synonyms: Orfadin® | SC-0735
Approved drug
nitisinone is an approved drug (FDA (2002), EMA (2005))
Compound class: Synthetic organic
Comment: Inhibits 4-hydroxyphenylpyruvate dioxygenase (HPD)
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2D Structure
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Physico-chemical Properties
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Hydrogen bond acceptors 3
Hydrogen bond donors 0
Rotatable bonds 4
Topological polar surface area 94.35
Molecular weight 329.05
XLogP 1.59
No. Lipinski's rules broken 0
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Canonical SMILES O=C1CCCC(=O)C1C(=O)c1ccc(cc1[N+](=O)[O-])C(F)(F)F
Isomeric SMILES O=C1CCCC(=O)C1C(=O)c1ccc(cc1[N+](=O)[O-])C(F)(F)F
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
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Summary of Clinical Use Click here for help
Used to treat hereditary tyrosinemia type 1, in combination with controlled dietary intake of tyrosine and phenylalanine. In the EU the EMA has granted orphan disease designation for this drug for alkaptonuria and tyrosinaemia type 1 .
Mechanism Of Action and Pharmacodynamic Effects Click here for help
A synthetic reversible inhibitor of 4-hydroxyphenylpyruvate dioxygenase
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