Nitisinone — Uses, Dosage, Side Effects & Safety

Drug class: Tyrosine metabolism inhibitor

Overview

Nitisinone is a prescription medicine used to treat hereditary tyrosinemia type 1 (HT-1), a rare genetic disorder that affects the breakdown of the amino acid tyrosine in the body. By blocking a specific enzyme, nitisinone helps prevent the buildup of toxic substances that can damage the liver, kidneys, and nervous system. Nitisinone is not a cure for HT-1, but it can significantly reduce symptoms and improve quality of life when used alongside a special diet low in tyrosine and phenylalanine. Early and consistent treatment is important to prevent serious complications. Patients should follow their healthcare provider’s instructions closely and attend regular check-ups to monitor their health while taking nitisinone.

Frequently Asked Questions about Nitisinone

What is nitisinone used for?

Nitisinone is used to treat hereditary tyrosinemia type 1 (HT-1), a rare genetic disorder that affects the breakdown of tyrosine, helping to prevent liver and kidney damage.

How does nitisinone work?

Nitisinone blocks an enzyme called 4-hydroxyphenylpyruvate dioxygenase, reducing the production of toxic substances that can harm the liver and kidneys.

What are the most common side effects?

Common side effects include increased tyrosine levels, eye pain or redness, skin rash, and headache.

Can nitisinone be used during pregnancy?

Nitisinone should only be used during pregnancy if clearly needed. Discuss the risks and benefits with your healthcare provider.

Do I need a special diet while taking nitisinone?

Yes, a diet low in tyrosine and phenylalanine is required to prevent complications. Your healthcare team will provide dietary guidance.

How should I store nitisinone?

Store at room temperature, away from moisture and heat. Follow the storage instructions on the packaging.

What should I do if I miss a dose?

Take the missed dose as soon as you remember, unless it is almost time for your next dose. Do not double up doses.

Educational information only. Always consult a qualified healthcare professional before starting, stopping, or changing any medication.