Nitisinone: Mechanism of Action

Comparator Drug Clinical Trial12 Jun, 2023Health

Nitisinone inhibits the tyrosine catabolic enzyme 4-hydroxyphenyl-pyruvate dioxygenase, which occurs before fumarylacetoacetate hydrolase (FAH). The HT-1 patients' accumulation of maleylacetoacetate and fumarylacetoacetate is reduced by nitisinone's impairment of tyrosine's regular metabolism. The toxic metabolites succinylacetone and succinyl acetoacetate that are created when these catabolic intermediates are converted in the body are what cause the liver and kidney damage that is observed in HT-1 patients. Additionally, succinylacetone can disrupt the pathway for porphyrin formation, which causes an accumulation of 5-aminolevulinate, a neurotoxin that triggers the porphyric crisis associated with HT-1. The drug can be acquired with a valid prescription; therapy should begin under a doctor's supervision; and the nitisinone price in India is affordable, so you can easily add it to your treatment.