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Nitisinone

Catalog No. DB00348 Name DrugBank
CAS Number 104206-65-7 Website http://www.ualberta.ca/
M. F. C14H10F3NO5 Telephone (780) 492-3111
M. W. 329.2281096 Fax (780) 492-1071
Purity Email david.wishart@ualberta.ca
Storage Chembase ID: 232

SYNONYMS

IUPAC name
2-[2-nitro-4-(trifluoromethyl)benzoyl]cyclohexane-1,3-dione
IUPAC Traditional name
2-[2-nitro-4-(trifluoromethyl)benzoyl]cyclohexane-1,3-dione
Brand Name
Orfadin
Synonyms
nitisinone

DATABASE IDS

CAS Number 104206-65-7
PubChem SID 46507380
PubChem CID 115355

PROPERTIES

Hydrophobicity(logP) 1.6

DETAILS

Description (English)
Item Information
Drug Groups approved; investigational
Description Nitisinone is a synthetic reversible inhibitor of 4-hydroxyphenylpyruvate dioxygenase. It is used in the treatment of hereditary tyrosinemia type 1. It is sold under the brand name Orfadin. [Wikipedia]
Indication Used as an adjunct to dietary restriction of tyrosine and phenylalanine in the treatment of hereditary tyrosinemia type 1.
Pharmacology Hereditary tyrosinemia type 1 occurs due to a deficiency in fumarylacetoacetase (FAH), the final enzyme in the tyrosine catabolic pathway. Nitisinone inhibits catabolism of tyrosine by preventing the catabolic intermediates. In patients with HT-1, these catabolic intermediates are converted to the toxic metabolites succinylacetone and succinylacetoacetate, which are responsible for the observed liver and kidney toxicity. Succinylacetone can also inhibit the porphyrin synthesis pathway leading to the accumulation of 5-aminolevulinate, a neurotoxin responsible for the porphyric crises characteristic of HT-1.
Toxicity Side effects include elevated plasma levels of this amino acid, hepatic and liver failure.
Affected Organisms
Humans and other mammals
Absorption The capsule and liquid formulations are bioequivalent in both the plasma concentration-time curve and maximum plasma concentration (Cmax).
Half Life ~54 hours
External Links
Wikipedia
RxList
Drugs.com

REFERENCES