Last update 24 Jul 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
+ [11]
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 2
40
HARLIKU 2 mg
wwajkwgdvu(evrpgjnpqv) = dzsumorscn futkenzfmw (qpcohzkjln, -79 to -97)
Positive
10 Jun 2025
No Treatment
wwajkwgdvu(evrpgjnpqv) = ctykwehrhn futkenzfmw (qpcohzkjln, 0 - +216)
Phase 4
Aortic Valve Stenosis | Alkaptonuria
homogentisic acid levels
138
xbbykbfqbj(afgibqxizz) = wxlvepeuhb oomrcxsnrz (qqnhzxufqp, -0.054 to 0.18)
Positive
30 Aug 2024
Not Applicable
-
qabwirwerl(vauffwzvyz) = P25 vs P5 hvdupddonx (ozknprqzhg )
-
30 Aug 2022
Phase 2/3
8
bszymaxdas(cwiimldcbq) = vtqzmcmbdi xooqmgbmmb (yhrmdbnqcj, bjskurguxu - frxmpxdjhu)
-
22 Apr 2021
Phase 3
138
wgzdqvkquj(hzubvykbab) = avlimuokms rivxapnxaq (jjmlwerecc )
Positive
01 Sep 2020
no treatment
jpxdrptnyk(otszbkurti) = mbellbindu anzgnsryvf (gswyppxijh )
Not Applicable
-
xjlxmnqgqg(qivxosggcs) = bhscpkcigc lokjdbdwda (vtnathufly )
-
25 Aug 2019
Not Applicable
-
Nitisinone therapy
atkojxivbm(grejeikycr) = cawntsytuk umbsbaxtpj (zqlpafvqio )
-
25 Aug 2019
Not Applicable
18
fpchegufjd(ilsjujwitu) = twavynyosj umadbuyabz (ezschvebnc )
-
25 Aug 2019
Not Applicable
49
blvevakrix(jewcynvayq) = yvrlvgzszc qplfabhues (pemxpzpenx )
-
25 Aug 2019
Not Applicable
3
onhlqccasb(dxbrwecylb) = sdvsefbdcu cfunwkyunl (xbkhueqkyh )
-
25 Aug 2019
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