Compound Detail

CHEMBL1337

NITISINONE

💊 Approved Drug

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💊 Approved Drug

O=C1CCCC(=O)C1C(=O)c1ccc(C(F)(F)F)cc1[N+](=O)[O-]

ChEMBL ↗ Properties Compare Identifier

Basic Information

ChEMBL ID	CHEMBL1337
Name	NITISINONE
Phase	Approved
Structure Type	MOL
InChI Key	OUBCNLGXQFSTLU-UHFFFAOYSA-N
Therapeutic	✓ Yes

Known Names

Nitisinona Nitisinone Nitisinone mdk (previously nitisinone mendelikabs) Nityr Orfadin SC-0735

Targets

Activities

Assay Types

PK Parameters

Publications

Known Names

Indications

Mechanisms

Molecular Properties

329.2

MW (Da)

2.73

ALogP

HBA

HBD

94.3

PSA (Å²)

0.37

QED

Ro5 Violations

Rot. Bonds

Drug Information

Molecule Type	Small molecule
First Approval	2002
Availability	Prescription
Chirality	Achiral

Routes of Administration

Oral

Flags

First in Class

USAN Year

2002

Extended Properties

Molecular Formula	C14H10F3NO5
MW	329.23
Aromatic Rings	1
Heavy Atoms	23
NP-Likeness	-1.05

Mechanism of Action

Mechanism	Action Type	Target	Direct	Efficacy
4-hydroxyphenylpyruvate dioxygenase inhibitor	INHIBITOR	4-hydroxyphenylpyruvate dioxygenase	✓	✓

Indications

Tyrosinemias Alkaptonuria Albinism

ATC Classification

A16AX04

nitisinone

Level 1: ALIMENTARY TRACT AND METABOLISM

Level 2: OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS

Activity Summary

IC50 7 meas. best 7.4

Ki 1 meas. best 7.43

Target Activity Profile

Target	Type	Best pChEMBL	Mean pChEMBL	Best (nM)	# Data
4-hydroxyphenylpyruvate dioxygenase CHEMBL1861	Ki	7.43	7.43	37.0	1
4-hydroxyphenylpyruvate dioxygenase CHEMBL3203	IC50	7.4	7.4	40.0	1
4-hydroxyphenylpyruvate dioxygenase CHEMBL5863	IC50	7.4	7.4	40.0	2
MC-38 CHEMBL612821	IC50	4.02	4.02	96000.0	1

Pharmacokinetic Data

Parameter	Value	Units	Species
T1/2	63.0	hr	Rattus norvegicus
T1/2	53.0	hr	Homo sapiens
T1/2	1.0	hr	Homo sapiens
T1/2	53.0	hr	Homo sapiens
T1/2	63.0	hr	Rattus norvegicus

Activity Data Samples

Top measurements by pChEMBL value

Target	Type	Rel.	Value	Units	pChEMBL	Assay	PubMed
4-hydroxyphenylpyruvate dioxygenase	Ki	=	37.0	nM	7.43	Inhibition of purified His6-tagged recombinant human HPPD assessed as inhibition...	25182962
4-hydroxyphenylpyruvate dioxygenase	IC50	=	40.0	nM	7.4	Inhibition of 4-hydroxyphenylpyruvate dioxygenase of purified pig liver by enol-...	10853644
4-hydroxyphenylpyruvate dioxygenase	IC50	=	40.0	nM	7.4	Inhibition of Wistar rat liver HPPD using 4-Hydroxyphenylpyruvate as substrate a...	28128559
4-hydroxyphenylpyruvate dioxygenase	IC50	=	40.0	nM	7.4	Inhibition of Wistar rat liver cytosol HPPD using HPP as substrate assessed as r...	28128559
MC-38	IC50	=	96000.0	nM	4.02	Antiproliferative activity against mouse MC-38 cells assessed as inhibition of c...	37983010

Literature References

PubMed	DOI	Target Context	# Activities
28128559	10.1021/acs.jmedchem.6b01395	Homo sapiens	6
37983010	10.1021/acs.jmedchem.3c00281	ADMET	5
37983010	10.1021/acs.jmedchem.3c00281	Fusobacterium nucleatum	4
34547896	10.1021/acs.jmedchem.1c00823	Transthyretin	4
28128559	10.1021/acs.jmedchem.6b01395	Rattus norvegicus	4
28128559	10.1021/acs.jmedchem.6b01395	4-hydroxyphenylpyruvate dioxygenase	4
-	10.6019/CHEMBL4495565	SARS-CoV-2	4
20014752	10.1021/tx900326k	Hepatotoxicity	3
-	10.6019/CHEMBL4495564	Replicase polyprotein 1ab	2
-	10.6019/CHEMBL4808148	Histone deacetylase 6	2
26948801	10.1016/j.drudis.2016.02.015	Homo sapiens	2
37983010	10.1021/acs.jmedchem.3c00281	Unchecked	2
-	10.1101/2020.04.21.054387	SARS-CoV-2	1
28128559	10.1021/acs.jmedchem.6b01395	Plasma	1
25182962	10.1016/j.bmc.2014.08.011	4-hydroxyphenylpyruvate dioxygenase	1
37983010	10.1021/acs.jmedchem.3c00281	MC-38	1
37983010	10.1021/acs.jmedchem.3c00281	HCT-116	1
37983010	10.1021/acs.jmedchem.3c00281	HUVEC	1
37983010	10.1021/acs.jmedchem.3c00281	L02	1
37983010	10.1021/acs.jmedchem.3c00281	Escherichia coli	1

Data from ChEMBL 36 via Core Engine