Nitisinone Capsules

Dosage form: capsule
Drug class:Miscellaneous metabolic agents

Medically reviewed by Drugs.com. Last updated on Jun 1, 2022.

Indications and Usage for Nitisinone Capsules

Nitisinone Capsules are indicated for the treatment of adult and pediatric patients with hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine.

Nitisinone Capsules Dosage and Administration

Dosage

Starting Dosage

The recommended starting dosage of Nitisinone Capsules is 0.5 mg/kg administered orally twice daily.

Maintenance Regimen

In patients 5 years of age and older who have undetectable serum and urine succinylacetone concentrations after a minimum of 4 weeks on a stable dosage of nitisinone, the total daily dose of Nitisinone Capsules may be given once daily (e.g., 1 to 2 mg/kg once daily) [see Clinical Pharmacology (12.2)].

Dosage Titration

Titrate the dosage in each individual patient based on biochemical and/or clinical response.

• Monitor plasma and/or urine succinylacetone concentrations, liver function parameters and alpha-fetoprotein levels.
• If succinylacetone is still detectable in blood or urine 4 weeks after the start of nitisinone treatment, increase the nitisinone dosage to 0.75 mg/kg twice daily. A maximum total daily dosage of 2 mg/kg may be needed based on the evaluation of all biochemical parameters.
• If the biochemical response is satisfactory (undetectable blood and/or urine succinylacetone), the dosage should be adjusted only according to body weight gain and not according to plasma tyrosine levels.
• During initiation of therapy, when switching from twice daily to once daily dosing, or if there is a deterioration in the patient’s condition, it may be necessary to follow all available biochemical parameters more closely (i.e. pla...