About nitisinone
Tyrosine metabolism inhibitor
Used for
- Hereditary tyrosinemia type 1 (HT-1)
- Alkaptonuria in adults
Usual adult dose
HT-1: usually 1 mg/kg daily, adjusted by specialist. Alkaptonuria: usually 10 mg once daily.
Usual dose in children
HT-1: usually 1 mg/kg daily, adjusted by specialist; dosing may be divided twice daily.
Common side effects
- Low white blood cell or platelet counts
- Eye inflammation or corneal changes
- Rash, itching, or hair loss
- Raised tyrosine levels, sometimes causing eye or skin problems
Important warnings
- HT-1 treatment requires dietary restriction of tyrosine and phenylalanine.
- Regular blood tests and eye checks are needed.
- Do not stop suddenly; tyrosine and related substances can rise dangerously.
- Use only under specialist supervision.
Pregnancy
Use only when clearly needed; untreated HT-1 can be dangerous. Specialist monitoring is important.
Breastfeeding
Breastfeeding is generally not recommended during treatment because effects on the infant are uncertain.
Interactions
- No major established drug interactions are known.
- Other medicines affecting tyrosine metabolism may require specialist review.
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 It may be incomplete or out of date.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.