Table of contents

  • Brand Names
  • Chemistry
  • Pharmacologic Category
  • Mechanism of Action
  • Therapeutic Use
  • Pregnancy and Lactation Implications
  • Contraindications
  • Warnings and Precautions
  • Adverse Reactions
  • Toxicological Effects
  • Genes that may be involved
  • Nutrition/Nutraceutical Interactions
  • Dosage
  • Pharmacokinetics and Pharmacodynamics
  • Special Considerations

Brand Names


Austria: Orfadin; Belgium: Orfadin; Bulgaria: Orfadin; Cyprus: Orfadin; Czech Republic: Orfadin; Denmark: Orfadin; Estonia: Orfadin; Finland: Orfadin; France: Orfadin; Germany: Orfadin; Greece: Orfadin; Hungary: Orfadin; Ireland: Orfadin; Italy: Orfadin; Latvia: Orfadin; Lithuania: Orfadin; Luxembourg: Orfadin; Malta: Orfadin; Netherlands: Orfadin; Poland: Orfadin; Portugal: Orfadin; Romania: Orfadin; Slovakia: Orfadin; Slovenia: Orfadin; Spain: Orfadin; Sweden: Orfadin; UK: Orfadin.

North America

USA: Orfadin.

Drug combinations


Nitisinone: C~14~H~10~F~3~NO~5~. Mw: 329.23. (1) 1,3-Cyclohexanedione, 2-[2-nitro-4-(trifluoromethyl)benzoyl]-; (2) 2-(α,α,α-Trifluoro-2-nitro-p-tuluoyl)-1,3-cyclohexanedione. CAS-104206-65-7 (2002).

Pharmacologic Category

Other Miscellaneous Therapeutic Agents; 4-Hydroxyphenylpyruvate Dioxygenase Inhibitor. (ATC-Code: A16AX04).

Mechanism of action

In hereditary tyrosinemia type 1 (HT-1), tyrosine metabolism is interrupted due to lack of enzyme (fumarylacetoacetate hydrolase) needed in last step of tyrosine degradation. Toxic metabolites of tyrosine accumulate and cause liver and kidney toxicity. Nitisinone competitively inhibits 4-hydroxyphenyl-pyruvate dioxygenase, enzyme needed earlier in tyrosine degradation pathway; therefore prevents build-up of damaging metabolites.

Therapeutic use

Treatment of hereditary tyrosinemia type 1. To be used with dietary restriction of tyrosine and phenylalanine.

Pregnancy and lactiation implications

Safety and efficacy not established for pregnant women. Use only if potential benefit to mother outweighs possible risk to fetus. Excretion in breast milk unknown (use caution).

Unlabeled use


Hypersensitivity to nitisinone or any component of the formulation.

Warnings and precautions

Transient leukopenia and/or thrombocytopenia may occur. Must be used with dietary restriction of tyrosine and phenylalanine (inadequate restriction can result in toxic effects to eyes, skin, and nervous system).



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