Nitisinone

Chemical formula: C₁₄H₁₀F₃NO₅  Molecular mass: 329.228 g/mol  PubChem compound: 115355

Active ingredient description

Nitisinone is a competitive inhibitor of 4-hydroxyphenylpyruvate dioxygenase, an enzyme which precedes fumarylacetoacetate hydrolase in the tyrosine catabolic pathway. By inhibiting the normal catabolism of tyrosine in patients with hereditary tyrosinemia type 1 (HT-1), nitisinone prevents the accumulation of the toxic intermediates maleylacetoacetate and fumarylacetoacetate.

Medicine classification

This medicinal substance has been classified in the anatomical therapeutic chemical (ATC) classification according to its main therapeutic use as follows:

ATC code Group title Classification
A16AX04 Nitisinone A Alimentary tract and metabolism → A16 Other alimentary tract and metabolism products → A16A Other alimentary tract and metabolism products → A16AX Various alimentary tract and metabolism products
Discover more medicines within A16AX04

Product monographs

Competent medicine agencies globally have authorized commercialization of this active ingredient according to these medication package inserts (MPIs):

Title Information Source Document Type  
ORFADIN Capsule / Oral suspension FDA, National Drug Code (US) MPI, US: SPL/PLR
NITISINONE MDK Hard capsule European Medicines Agency (EU) MPI, EU: SmPC

Structural formula

Graphic representation of the active ingredient's molecular structure

External identifiers

CAS Substance: 104206-65-7
DrugBank Drug: DB00348
PubChem Compound: 115355
RxNorm Ingredient: 61805
SNOMED-CT Concept: 385996000
Nitisinone (substance)
UNII Identifier: K5BN214699
NITISINONE

Medicines

Nitisinone is an active ingredient of these brands:

United States (US)

Austria (AT)

Brazil (BR)

Canada (CA)

Croatia (HR)

Estonia (EE)

Finland (FI)

France (FR)

Ireland (IE)

Israel (IL)

Italy (IT)

Japan (JP)

Lithuania (LT)

Netherlands (NL)

New Zealand (NZ)

Poland (PL)

Romania (RO)

Spain (ES)

Tunisia (TN)

Turkey (TR)

United Kingdom (UK)

Note the following: The list of brand names is continuously updated, and thus does not include the total of products circulating worldwide.

 
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