Nitisinone Brand Names- NITYR | Orfadin
What is Nitisinone
Nitisinone inhibits the breakdown of tyrosine in patients with hereditary tyrosinemia type I (HT-1).
Patients with HT-1 are deficient in fumarylacetoacetate hydrolase (FAH), the final enzyme in the tyrosine catabolism pathway.
Fewer than 100 children in the US are affected by this condition. Nitisinone was originally developed as a weed killer, but was unsuccessful. Swedish scientists then discovered its use in the treatment of HT-1.
Prior to the introduction of nitisinone, the only treatments for HT-1 were dietary restriction of tyrosine and phenylalanine and liver transplantation.
The 2- and 4-year survival probabilities for patients presenting with HT-1 under 2 months of age and treated with dietary restriction and nitisinone are 88% and 88%, respectively, as compared to historical controls treated with dietary restriction alone, 29% and 29%, respectively.
The clinical trial of nitisinone also suggests a decreased incidence of porphyric crisis in these patients.
Because nitisinone inhibits the breakdown of tyrosine, treatment with nitisinone requires restriction of dietary intake of tyrosine and phenylalanine to prevent toxicity.
During dosage titration, monitor plasma and/or urine succinylacetone concentrations, liver function parameters, and alpha-fetoprotein levels. Due to the potential for thrombocytopenia or leukopenia, monitor platelet and white blood cell counts during therapy.
- hereditary tyrosinemia type I
For the treatment of hereditary tyrosinemia type I
NOTE: Nitisinone has been designated an orphan drug by the FDA for this indication.
- abdominal pain
- corneal opacification
- exfoliative dermatitis
- hepatic failure
- maculopapular rash
- ocular pain
- serum alpha-fetoprotein
- serum phenylalanine (Phe) concentrations
- serum tyrosine
- urinary succinylacetone
- hepatic disease
- neoplastic disease
- ocular disease
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