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Nitisinone

FDA-approved product (capsule, oral), marketed as Harliku, Nitisinone, Nityr and 1 more.

Registered studies
17
Lab records
0
Pathogens at ≥40%
1 of 4

Chemical structure

Chemical structure of Nitisinone
A drawing shows what the molecule is, not what it does.

Existing / approved use

Documented · FDA, WHO, ChEMBL

Approved for

  • Tyrosinemia

Indications ChEMBL records as approved, from FDA and DailyMed labels.

WHO therapeutic group
Various alimentary tract and metabolism products
FDA pharmacologic class
4-Hydroxyphenyl-Pyruvate Dioxygenase Inhibitor FDA label ↗
Anti-infective classification
Not classified as an anti-infective.

Repurposing investigation

This medicine is being investigated here for AI-predicted antimicrobial activity against M. tuberculosis. It was surfaced computationally for further investigation; it is not an established treatment for this infection.

AI-predicted activity

AI prediction · nothing measured
  • MRSA32.5% AI-predicted activityBelow the ≥40% mark this site uses to surface candidates.
  • E. coli24.2% AI-predicted activityBelow the ≥40% mark this site uses to surface candidates.
  • K. pneumoniae24.0% AI-predicted activityBelow the ≥40% mark this site uses to surface candidates.
  • M. tuberculosis59.6% AI-predicted activityAt or above the ≥40% mark this site uses to surface candidates.

A model’s estimate of laboratory activity against each pathogen species. It is not clinical effectiveness.

Evidence

Clinical and experimental

Documented evidence

Clinical

17registered studies name this medicine, for any condition. See them below.

Experimental

No evidence found

No laboratory measurement against the four pathogens in the ChEMBL records loaded here.

Docking

Computational · nothing measured

Dihydrofolate reductase ↗ (E. coli)

-7.4 kcal/mol

0better fit →-12

KPC-2 carbapenemase ↗ (K. pneumoniae)

-8.8 kcal/mol

0better fit →-12

Enoyl-ACP reductase (InhA) ↗ (M. tuberculosis)

-8.2 kcal/mol

0better fit →-12

Dihydrofolate reductase ↗ (MRSA)

-9.0 kcal/mol

0better fit →-12

AutoDock Vina, one experimentally solved protein per pathogen. This project’s screening target is -7.0 kcal/mol, its own mark rather than a universal cutoff. A docking score is a structural hypothesis, not proof of binding, and requires experimental validation.

Other medicines to investigate

AI prediction · nothing measured

Other repurposing candidates: approved medicines besides Nitisinone with AI-predicted activity ≥40% against the chosen pathogen, leaving out existing antimicrobials. They are computational candidates for further investigation, not alternatives and not recommendations.

Sorted by AI-predicted activity, high to low. The order is not a ranking of which medicine is better.

Registered studies

A registration describes a study; it does not establish a positive result.

  • Long-Term Study of Nitisinone to Treat Alkaptonuria

    NCT00107783 · start 2005-01

    Registered record
    Registry ID
    NCT00107783
    Conditions
    Alkaptonuria
    Interventions
    Nitisinone (NTBC)
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2005-01
    Completion
    2009-04
    Enrolment
    40

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionAlkaptonuria
    InterventionNitisinone (NTBC)
    PhasePhase 2
    Registry statusCompleted
  • Prevention of Dichloroacetate Toxicity

    NCT00031161 · start 2001-09

    Registered record
    Registry ID
    NCT00031161
    Conditions
    Acidosis, Lactic; Chronic Disease
    Interventions
    Nitisinone (NTBC); Dichloroacetate; Low-tyrosin diet
    Study type
    INTERVENTIONAL
    Phase
    Not applicable
    Registry status
    Completed
    Start
    2001-09
    Completion
    2005-09
    Enrolment
    30

    As stored from ClinicalTrials.gov. Registration describes a study; it does not mean the study worked.

    ConditionAcidosis, Lactic; Chronic Disease
    InterventionNitisinone (NTBC); Dichloroacetate; Low-tyrosin diet
    PhaseNot applicable
    Registry statusCompleted