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Orfadin Brand names, Orfadin Analogs

Orfadin Brand Names Mixture

  • No information avaliable

Orfadin Chemical_Formula


Orfadin RX_link


Orfadin fda sheet

Orfadin FDA

Orfadin msds (material safety sheet)

Orfadin Synthesis Reference

No information avaliable

Orfadin Molecular Weight

329.228 g/mol

Orfadin Melting Point

No information avaliable

Orfadin H2O Solubility

No information avaliable

Orfadin State

No information avaliable

Orfadin LogP


Orfadin Dosage Forms

Capsules; Liquid

Orfadin Indication

Used as an adjunct to dietary restriction of tyrosine and phenylalanine in the treatment of hereditary tyrosinemia type 1.

Orfadin 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.

Orfadin Absorption

The capsule and liquid formulations are bioequivalent in both the plasma concentration-time curve and maximum plasma concentration (Cmax).

Orfadin side effects and Toxicity

Side effects include elevated plasma levels of this amino acid, hepatic and liver failure.

Orfadin Patient Information

Orfadin Organisms Affected

Humans and other mammals