About nitisinone
Tyrosine metabolism inhibitor
Used for
- Hereditary tyrosinemia type 1, alongside dietary restriction.
- Alkaptonuria in adults.
Usual adult dose
Tyrosinemia: usually 1–2 mg/kg daily, adjusted by blood tests. Alkaptonuria: 10 mg once daily.
Usual dose in children
Tyrosinemia: usually 1–2 mg/kg daily, adjusted by blood tests. Not used for alkaptonuria in children.
Common side effects
- Low blood platelet or white blood cell counts.
- Eye irritation, light sensitivity, or corneal changes.
- Rash, itching, or hair loss.
- Raised liver enzymes or abnormal blood tests.
Important warnings
- Tyrosinemia treatment requires a restricted tyrosine and phenylalanine diet.
- Regular blood tests and eye examinations are needed.
- Report eye pain, redness, vision changes, or unusual bruising promptly.
- Use under specialist supervision; dose may need adjustment.
Pregnancy
Use only when clearly needed; untreated tyrosinemia also carries serious risks. Specialist monitoring is important.
Breastfeeding
Breastfeeding is generally not recommended during treatment because infant safety is uncertain.
Interactions
- Medicines that inhibit CYP2C9, such as fluconazole, may increase nitisinone levels.
- Medicines that induce CYP3A4, such as rifampicin, may lower nitisinone levels.
AI-generated from general medical knowledge, not from this product's official leaflet. It may be incomplete or out of date. Not medical advice — ask a pharmacist or doctor before making any decision.