Nitisinone

4-Hydroxyphenyl-Pyruvate Dioxygenase Inhibitor — ALIMENTARY TRACT AND METABOLISM

Key facts

Nitisinone is an ofp drug in the 4-Hydroxyphenyl-Pyruvate Dioxygenase Inhibitor class catalogued in DrugCentral. It was first approved by the FDA in 2002. Its mechanism of action is classified as Cytochrome P450 2C9 Inhibitors, Cytochrome P450 2E1 Inducers and Hydroxyphenylpyruvate Dioxygenase Inhibitors. DrugCentral lists 3 molecular targets, including HPD, Hpd and HPD. Recorded indications include Homogentisate 1,2dioxygenase deficiency and Tyrosinemia type I.

• Approval status: ofp (first approved 2002 by the FDA).
• Drug class: 4-Hydroxyphenyl-Pyruvate Dioxygenase Inhibitor.
• Mechanism of action: Cytochrome P450 2C9 Inhibitors; Cytochrome P450 2E1 Inducers; Hydroxyphenylpyruvate Dioxygenase Inhibitors.
• Molecular targets: HPD, Hpd and HPD (3 listed in DrugCentral).
• Indications: Homogentisate 1,2dioxygenase deficiency and Tyrosinemia type I.
• ATC code: A16AX04 (Various alimentary tract and metabolism products).
• Molecular formula: C14H10F3NO5; molecular weight 329.23 g/mol.

Indications & contraindications

Drug–disease relationships curated by DrugCentral (OMOP / SNOMED concepts).

Indications (2)

• Homogentisate 1,2dioxygenase deficiency
• Tyrosinemia type I

Contraindications (8)

• Hypertyrosinemia
• Hypophosphatemia
• Kidney disease
• Leukopenia
• Pain in eye
• Photophobia
• Red eye
• Thrombocytopenic disorder

Mechanism & targets

• Mechanism of action (MoA): Cytochrome P450 2C9 Inhibitors; Cytochrome P450 2E1 Inducers; Hydroxyphenylpyruvate Dioxygenase Inhibitors; Organic Anion Transporter 1 Inhibitors; Organic Anion Transporter 3 Inhibitors
• Established pharmacologic class: 4-Hydroxyphenyl-Pyruvate Dioxygenase Inhibitor

Molecular targets (3)

Target	Gene	Action	Activity
4-hydroxyphenylpyruvate dioxygenase	HPD	unknown	IC50 7.40
4-hydroxyphenylpyruvate dioxygenase	Hpd	unknown	IC50 7.40
4-hydroxyphenylpyruvate dioxygenase	HPD	inhibitor	IC50 7.40

Regulatory approvals (2)

Date	Agency	Marketed as	Applicant	Orphan
2005-02-21	EMA	—	Swedish Orphan Biovitrum International AB
2002-01-18	FDA	—	SWEDISH ORPHAN	Yes

ATC classification

ATC code	Therapeutic hierarchy
A16AX04	ALIMENTARY TRACT AND METABOLISM › OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS › OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS › Various alimentary tract and metabolism products

Reported adverse-event signals

Strongest FDA FAERS disproportionality signals (log-likelihood ratio). A signal is a statistical association in spontaneous reports, not proof that the drug causes the event.

MedDRA term	LLR
Amino acid level increased	856.8
Liver transplant	497.4
Succinylacetone increased	348.6
Hepatocellular carcinoma	319.8
Alpha 1 foetoprotein increased	284.7
Lenticular opacities	262.7
Cataract	145.7
Amino acid level decreased	135.1
Treatment noncompliance	115.3
Hepatic failure	95.6

Chemical structure

• Molecular formula: C₁₄H₁₀F₃NO₅
• Molecular weight: 329.23 g/mol
• CAS number: —
• InChIKey: OUBCNLGXQFSTLU-UHFFFAOYSA-N
• PubChem CID: 115355

[O-][N+](=O)C1=C(C=CC(=C1)C(F)(F)F)C(=O)C1C(=O)CCCC1=O

Frequently asked questions

Answers are generated from the structured data on this page, not written by hand.

What is Nitisinone used for?

DrugCentral lists the following indications for Nitisinone: Homogentisate 1,2dioxygenase deficiency and Tyrosinemia type I.

How does Nitisinone work?

Nitisinone is classified as Cytochrome P450 2C9 Inhibitors, Cytochrome P450 2E1 Inducers and Hydroxyphenylpyruvate Dioxygenase Inhibitors; its molecular targets in DrugCentral include HPD, Hpd and HPD (inhibitor).

When was Nitisinone first approved?

Nitisinone was first approved by the FDA in 2002.

What drug class does Nitisinone belong to?

Nitisinone is in the 4-Hydroxyphenyl-Pyruvate Dioxygenase Inhibitor class.

What is the chemical formula of Nitisinone?

Nitisinone has the molecular formula C14H10F3NO5 and a molecular weight of 329.23 g/mol.

Sources

• DrugCentral drug card 1944 — structure, indications, targets, approvals, pharmacologic classes (CC BY-SA 4.0)
• ChEMBL CHEMBL1337 — compound record and bioactivity
• PubChem CID 115355 — chemical identifiers
• DrugBank DB00348 — drug record
• FDA UNII K5BN214699 — substance identity
• DailyMed: FDA labels for Nitisinone — current prescribing information (label search)
• UniProt P32754 (HPD) — target protein
• FDA Adverse Event Reporting System (openFDA FAERS) — adverse-event signals, as aggregated by DrugCentral

How this page is built: methodology · editorial policy & corrections.