• # LGM Pharma is a Nitisinone CAS# 104206-65-7 API supplier distributor based in the USA. Inquire about DMF, cGMP, price, availability, samples, sourcing, purity and more.
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Product Details:

  • Product Name: Nitisinone
  • CAS #: 104206-65-7
  • 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
  • InChl: 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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On July 27, 2020, LGM Pharma announced its acquisition of the formulation development and drug product contract manufacturing business of Nexgen Pharma, Inc. As a result, you will notice our new logo and visuals throughout the website. We’re working on updates to reflect the exciting, expanded CDMO capabilities and services we now can offer you.

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