Nitisinone

Names

[ Name ]:
Nitisinone

[Synonym ]:
nitisinonum
2-(2-Nitro-4-trifluoromethylbenzoyl)-1,3-cyclohexanedione
2-[2-Nitro-4-(trifluoromethyl)benzoyl]-1,3-cyclohexanedione
2-(2-nitro-4-(trifluoromethyl)benzoyl)cyclohexane-1,3-dione
Orfadin
Nitisinone [INN]
nitisinona
Orfadin (TN)
1,3-Cyclohexanedione, 2-[2-nitro-4-(trifluoromethyl)benzoyl]-
Nitisone
2-(2-Nitro-4-trifluoromethylbenzoyl)cyclohexane-1,3-dione
2-[2-nitro-4-(trifluoromethyl)benzoyl]cyclohexane-1,3-dione
Nitisinone

Biological Activity

[Description]:

Nitisinone(SC0735) is an inhibitor of the enzyme 4-hydroxyphenylpyruvate dioxygenase.Target: 4-Hydroxyphenylpyruvate DioxygenaseNitisinone is a drug used to slow the effects of hereditary tyrosinemia type 1. Nitisinone reduced urinary HGA levels from an average of 4.0 +/- 1.8 (SD) g/day to 0.2 +/- 0.2 g/day ( P < .001). Low-dose nitisinone effectively reduced urinary HGA levels in patients with alkaptonuria. Future long-term clinical trials are planned to determine the benefits of nitisinone in preventing joint deterioration and providing pain relief, and its long-term side effects [1]. Nitisinone can prevent the development of liver disease and significantly reduce the risk of developing hepatocellular carcinoma; however, vigorous surveillance for the development of HCC needs to be continued lifelong [2].

[Related Catalog]:

Signaling Pathways >> Others >> Others

Research Areas >> Metabolic Disease

[References]

[1]. Suwannarat, P., et al., Use of nitisinone in patients with alkaptonuria. Metabolism, 2005. 54(6): p. 719-28.

[2]. Santra, S. and U. Baumann, Experience of nitisinone for the pharmacological treatment of hereditary tyrosinaemia type 1. Expert Opin Pharmacother, 2008. 9(7): p. 1229-36.

[Related Small Molecules]

Chemical & Physical Properties

[ Density]:
1.5±0.1 g/cm3

[ Boiling Point ]:
486.2±45.0 °C at 760 mmHg

[ Melting Point ]:
129-131°C

[ Molecular Formula ]:
C₁₄H₁₀F₃NO₅

[ Molecular Weight ]:
329.228

[ Flash Point ]:
247.9±28.7 °C

[ Exact Mass ]:
329.051117

[ PSA ]:
97.03000

[ LogP ]:
1.37

[ Vapour Pressure ]:
0.0±1.2 mmHg at 25°C

[ Index of Refraction ]:
1.535

[ Storage condition ]:
2-8°C

MSDS

Click to get detail MSDS

Toxicological Information

CHEMICAL IDENTIFICATION

RTECS NUMBER :: GV0766666

CHEMICAL NAME :: 1,3-Cyclohexanedione, 2-(2-nitro-4-trifluorobenzoyl)-

CAS REGISTRY NUMBER :: 104206-65-7

LAST UPDATED :: 199806

DATA ITEMS CITED :: 1

HEALTH HAZARD DATA

ACUTE TOXICITY DATA

TYPE OF TEST :: TDLo - Lowest published toxic dose

ROUTE OF EXPOSURE :: Oral

SPECIES OBSERVED :: Rodent - rat

DOSE/DURATION :: 4200 ug/kg/6W-I

TOXIC EFFECTS :: Sense Organs and Special Senses (Eye) - corneal damage Blood - other changes

REFERENCE :: TXAPA9 Toxicology and Applied Pharmacology. (Academic Press, Inc., 1 E. First St., Duluth, MN 55802) V.1- 1959- Volume(issue)/page/year: 141,439,1996

Safety Information

[ Hazard Codes ]:
Xi

[ RIDADR ]:
NONH for all modes of transport

[ HS Code ]:
2914700090

Customs

[ HS Code ]: 2914700090

[ Summary ]:
HS: 2914700090 halogenated, sulphonated, nitrated or nitrosated derivatives of ketones and quinones, whether or not with other oxygen function Tax rebate rate:9.0% Supervision conditions:none VAT:17.0% MFN tariff:5.5% General tariff:30.0%

Articles

Generation of healthy mice from gene-corrected disease-specific induced pluripotent stem cells.

PLoS Biol. 9(7) , e1001099, (2011)

Using the murine model of tyrosinemia type 1 (fumarylacetoacetate hydrolase [FAH] deficiency; FAH⁻/⁻ mice) as a paradigm for orphan disorders, such as hereditary metabolic liver diseases, we evaluated...

Serum markers in alkaptonuria: simultaneous analysis of homogentisic acid, tyrosine and nitisinone by liquid chromatography tandem mass spectrometry.

Ann. Clin. Biochem. 52 , 597-605, (2015)

Alkaptonuria is a rare debilitating autosomal recessive disorder of tyrosine metabolism, where deficiency of homogentisate 1,2-dioxygenase results in increased homogentisic acid. Homogentisic acid is ...

A misguided 'pill in the pocket' approach with flecainide leading to cardiac arrest.

BMJ Case Rep. 2012 , doi:10.1136/bcr-2012-006868, (2012)

Related Compounds

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