Product Name

Nitisinone

CAS Number

104206-65-7

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Product Name:
Nitisinone
CAS Number:
104206-65-7
Indication:
Used as an adjunct to dietary restriction of tyrosine and phenylalanine in the treatment of hereditary tyrosinemia type 1.
Mode of Action:

Nitisinone is a competitive inhibitor of 4-hydroxyphenyl-pyruvate dioxygenase, an enzyme upstream of fumarylacetoacetate hydrolyase (FAH) in the tyrosine catabolic pathway. By inhibiting the normal catabolism of tyrosine in patients with hereditary tyrosinemia type 1 (HT-1), nitisinone prevents the accumulation of the catabolic intermediates maleylacetoacetate and fumarylacetoacetate.

Pharmacodynamics:

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.

IUPAC:
2-[2-nitro-4-(trifluoromethyl)benzoyl]cyclohexane-1,3-dione
ATC:
A16AX04 — Nitisinone
PubChem:
46507380
Formula:
C14-H10-F3-NO5
Molecular Mass:
Average: 329.2281 Monoisotopic: 329.05110705
Synonyms:
2-(alpha,alpha,alpha-Trifluoro-2-nitro-p-tuluoyl)-1,3-cyclohexanedione
SMILES:
[O-][N+](=O)C1=C(C=CC(=C1)C(F)(F)F)C(=O)C1C(=O)CCCC1=O
AHFS Code:
92:92.00 — Other Miscellaneous Therapeutic Agents
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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