Nitisinone

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Molecule Structure

Scientific Name

Nitisinone

Description of the Drug

Nitisinone is a hydroxyphenylpyruvate dioxygenase inhibitor used as an adjunct to dietary restrictions for the treatment of hereditary tyrosinemia type 1 (HT-1), which causes intolerance to tyrosine containing foods.

Source: DrugBank Online – DrugBank.com. Retrieved 2023-03-10 from
http://www.drugbank.ca/drugs/DB00348

Brand Name(s)

Nityr, Orfadin

Company Owner(s)

Novitium Pharma Llc, Medunik Canada Inc, Cycle Pharmaceuticals Ltd, Swedish Orphan Biovitrum Ab Publ

Mechanism(s) Of Action

Target Name Target Type Action Type Target ChEMBL ID
4-hydroxyphenylpyruvate dioxygenase SINGLE PROTEIN INHIBITOR CHEMBL1861

Unichem Links

SureChEMBL SCHEMBL338795
PharmGKB PA164777037
Human Metabolome Database HMDB0014492
DrugBank DB00348
PubChem: Thomson Pharma 14753104
PubChem 115355
Mcule MCULE-6109734568
BindingDB 50088804
EPA CompTox Dashboard DTXSID9042673
DrugCentral 1944
Brenda 219880 219930 219883 135218 151008 23285
ChemicalBook CB61386686
Guide to Pharmacology 6834
rxnorm NITISINONE ORFADIN NITYR
ChEBI 50378
ZINC ZINC000100014475