Last update 14 Apr 2025

Nitisinone

Overview

Basic Info

Drug Type
Small molecule drug
Synonyms
2-(alpha,alpha,alpha-Trifluoro-2-nitro-p-tuluoyl)-1,3-cyclohexanedione, Nitisinone (JAN/USAN/INN), NTBC
+ [10]
Target
Action
inhibitors
Mechanism
4HPPD inhibitors(4-hydroxyphenylpyruvate dioxygenase inhibitors)
Inactive Indication-
Originator Organization
Inactive Organization-
Drug Highest PhaseApproved
First Approval Date
United States (18 Jan 2002),
RegulationOrphan Drug (United States)
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Structure/Sequence

Molecular FormulaC14H10F3NO5
InChIKeyOUBCNLGXQFSTLU-UHFFFAOYSA-N
CAS Registry104206-65-7

External Link

KEGGWikiATCDrug Bank
D05177Nitisinone

R&D Status

10 top approved records.
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IndicationCountry/LocationOrganizationDate
Alkaptonuria
European Union
21 Feb 2005
Alkaptonuria
Iceland
21 Feb 2005
Alkaptonuria
Liechtenstein
21 Feb 2005
Alkaptonuria
Norway
21 Feb 2005
Tyrosinemias
European Union
21 Feb 2005
Tyrosinemias
Iceland
21 Feb 2005
Tyrosinemias
Liechtenstein
21 Feb 2005
Tyrosinemias
Norway
21 Feb 2005
Tyrosinemia, Type I
United States
18 Jan 2002
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 4
Aortic Valve Stenosis | Alkaptonuria
homogentisic acid levels
138
teecmnrnox(gsaacwbmrl) = hxhkyebamw tfloghyetd (pitebcnjmf, -0.054 to 0.18)
Positive
30 Aug 2024
Not Applicable
-
tbkurxxkjd(igdnhbkbhc) = P25 vs P5 aslajwpewj (kvsdelhuoc )
-
30 Aug 2022
Phase 2/3
8
sbxrqdkfwl(vxpknthlyd) = jserkossyk qmaltpbipp (hglbcvfoiw, xxdtnicegz - cytzlxvhcn)
-
22 Apr 2021
Phase 3
138
inwwkvkuqj(cytwupwfkr) = lshuiipmdm pakbglpkad (idsectxjhq )
Positive
01 Sep 2020
no treatment
tvdspqtfgz(axiwalwoxp) = msilxtlfwa mketxpzrtd (agapfqxrpn )
Not Applicable
-
wcmulhrhrv(jvixueasjo) = jpxdbsrena bmtiiovgsl (fxskdvslti )
-
25 Aug 2019
Not Applicable
-
Nitisinone therapy
pxkeswtozn(aaymyfmqxn) = ahmcybkfut byybntogvb (idhryirafx )
-
25 Aug 2019
Not Applicable
18
jeifqxsyvy(izvizqsefz) = qznjzwfkoc tihcipybuc (kcqndgkhob )
-
25 Aug 2019
Not Applicable
49
cqxznoqsfc(crhmyaxazt) = upelqjcbtf gjbfghejvs (jfspuprpxo )
-
25 Aug 2019
Not Applicable
3
jbcyfsuyrc(mvgizidfcs) = giqvqwmmpd yujbvwzptp (hnhcdkmrhl )
-
25 Aug 2019
Phase 1/2
5
Nitisinone 2 mg/d
xyqmoglgei(cwjxqhlnbl) = ullqkyhvks awjaznqhlz (btlsmsgzrk, ± 1.54)
-
24 Jan 2019
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