Skip to content
AMR Drug RepurposingSmart Screeningresearch prototype

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.

Open Nitisinone in ChEMBL Registered studies below

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.

  • A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients

    NCT06227429 · start 2025-09

    Registered record
    Registry ID
    NCT06227429
    Conditions
    Hereditary Tyrosinemia, Type I
    Interventions
    Nitisinone
    Study type
    OBSERVATIONAL
    Phase
    Not recorded
    Registry status
    Withdrawn
    Start
    2025-09
    Completion
    2027-06-30
    Enrolment
    0

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

    ConditionHereditary Tyrosinemia, Type I
    InterventionNitisinone
    PhaseNot recorded
    Registry statusWithdrawn

  • Bio Equivalency 20 Mgm Orfadin and 20 Mgm of Nitisonine

    NCT04113772 · start 2019-11-01

    Registered record
    Registry ID
    NCT04113772
    Conditions
    Hereditary Tyrosinemia, Type I
    Interventions
    Nitisinone; Orfadin
    Study type
    INTERVENTIONAL
    Phase
    Not applicable
    Registry status
    Unknown
    Start
    2019-11-01
    Completion
    2021-03-31
    Enrolment
    4

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

    ConditionHereditary Tyrosinemia, Type I
    InterventionNitisinone; Orfadin
    PhaseNot applicable
    Registry statusUnknown

  • A Study to Investigate the Potential Influence of Nitisinone on the Metabolism and the Transport of Other Drugs in Healthy Volunteers

    NCT03103568 · start 2017-03-28

    Registered record
    Registry ID
    NCT03103568
    Conditions
    Drug Drug Interaction
    Interventions
    Nitisinone in Arm A; Tolbutamide; Metoprolol; Chlorzoxazone; Furosemide; Nitisinone in Arm B
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2017-03-28
    Completion
    2017-07-24
    Enrolment
    36

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

    ConditionDrug Drug Interaction
    InterventionNitisinone in Arm A; Tolbutamide; Metoprolol; Chlorzoxazone +2 more
    PhasePhase 1
    Registry statusCompleted

  • Bioequivalence Study of Two Oral Nitisinone Formulations to Treat Hereditary Tyrosinemia (HT-1)

    NCT02750345 · start 2016-03

    Registered record
    Registry ID
    NCT02750345
    Conditions
    Hereditary Tyrosinemia, Type I
    Interventions
    Nitisinone; Nitisinone Baked Tablet; Orfadin
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2016-03
    Completion
    2016-05
    Enrolment
    24

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

    ConditionHereditary Tyrosinemia, Type I
    InterventionNitisinone; Nitisinone Baked Tablet; Orfadin
    PhasePhase 1
    Registry statusCompleted

  • Bioavailability Food-Effect Study of an Oral Nitisinone Formulation to Treat Hereditary Tyrosinemia (HT-1)

    NCT02750332 · start 2015-11

    Registered record
    Registry ID
    NCT02750332
    Conditions
    Hereditary Tyrosinemia, Type I
    Interventions
    Nitisinone
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2015-11
    Completion
    2016-01
    Enrolment
    20

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

    ConditionHereditary Tyrosinemia, Type I
    InterventionNitisinone
    PhasePhase 1
    Registry statusCompleted

  • Bioequivalence Study of Two Nitisinone Formulations Compared to Orfadin

    NCT02750709 · start 2015-10

    Registered record
    Registry ID
    NCT02750709
    Conditions
    Hereditary Tyrosinemia, Type I
    Interventions
    Nitisinone; Nitisinone 10 mg Tablet High Compritol; Orfadin
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2015-10
    Completion
    2016-01
    Enrolment
    24

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

    ConditionHereditary Tyrosinemia, Type I
    InterventionNitisinone; Nitisinone 10 mg Tablet High Compritol; Orfadin
    PhasePhase 1
    Registry statusCompleted

  • Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1

    NCT02323529 · start 2014-12

    Registered record
    Registry ID
    NCT02323529
    Conditions
    Hereditary Tyrosinemia, Type I
    Interventions
    Nitisinone
    Study type
    INTERVENTIONAL
    Phase
    Phase 3
    Registry status
    Completed
    Start
    2014-12
    Completion
    2015-09
    Enrolment
    18

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

    ConditionHereditary Tyrosinemia, Type I
    InterventionNitisinone
    PhasePhase 3
    Registry statusCompleted

  • Suitability of Nitisinone in Alkaptonuria 2

    NCT01916382 · start 2014-04

    Registered record
    Registry ID
    NCT01916382
    Conditions
    Alkaptonuria
    Interventions
    Nitisinone
    Study type
    INTERVENTIONAL
    Phase
    Phase 3
    Registry status
    Unknown
    Start
    2014-04
    Completion
    2020-02-02
    Enrolment
    140

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

    ConditionAlkaptonuria
    InterventionNitisinone
    PhasePhase 3
    Registry statusUnknown

  • Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care

    NCT02320084 · start 2013-09

    Registered record
    Registry ID
    NCT02320084
    Conditions
    Hereditary Tyrosinemia, Type I
    Interventions
    Nitisinone
    Study type
    OBSERVATIONAL
    Phase
    Not recorded
    Registry status
    Completed
    Start
    2013-09
    Completion
    2019-09-30
    Enrolment
    315

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

    ConditionHereditary Tyrosinemia, Type I
    InterventionNitisinone
    PhaseNot recorded
    Registry statusCompleted

  • Dose Response Study of Nitisinone in Alkaptonuria

    NCT01828463 · start 2013-05

    Registered record
    Registry ID
    NCT01828463
    Conditions
    Alkaptonuria
    Interventions
    Nitisinone
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2013-05
    Completion
    2013-12
    Enrolment
    40

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

    ConditionAlkaptonuria
    InterventionNitisinone
    PhasePhase 2
    Registry statusCompleted

  • Bioequivalence of Orfadin 20 mg Compared to Orfadin 10 mg Capsules.

    NCT01857362 · start 2013-05

    Registered record
    Registry ID
    NCT01857362
    Conditions
    Healthy
    Interventions
    Nitisinone 20 mg; Nitisinone 10 mg
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2013-05
    Completion
    2013-06
    Enrolment
    12

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

    ConditionHealthy
    InterventionNitisinone 20 mg; Nitisinone 10 mg
    PhasePhase 1
    Registry statusCompleted

  • Nitisinone for Type 1B Oculocutaneous Albinism

    NCT01838655 · start 2013-04-16

    Registered record
    Registry ID
    NCT01838655
    Conditions
    Albinism; Vision Loss
    Interventions
    Nitisinone
    Study type
    INTERVENTIONAL
    Phase
    Phase 1 / Phase 2
    Registry status
    Completed
    Start
    2013-04-16
    Completion
    2017-02-07
    Enrolment
    5

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

    ConditionAlbinism; Vision Loss
    InterventionNitisinone
    PhasePhase 1 / Phase 2
    Registry statusCompleted

  • Taste and Palatability of Orfadin Suspension

    NCT01734889 · start 2012-10

    Registered record
    Registry ID
    NCT01734889
    Conditions
    Hereditary Tyrosinemia, Type I
    Interventions
    Nitisinone
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2012-10
    Completion
    2013-03
    Enrolment
    18

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

    ConditionHereditary Tyrosinemia, Type I
    InterventionNitisinone
    PhasePhase 1
    Registry statusCompleted

  • Bioequivalence of Orfadin Suspension Compared to Orfadin Capsules, and the Effect of Food on the Bioavailability of the Suspension

    NCT01682538 · start 2012-08

    Registered record
    Registry ID
    NCT01682538
    Conditions
    Healthy Volunteers
    Interventions
    Nitisinone, capsule; Nitisinone, suspension
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2012-08
    Completion
    2012-10
    Enrolment
    12

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

    ConditionHealthy Volunteers
    InterventionNitisinone, capsule; Nitisinone, suspension
    PhasePhase 1
    Registry statusCompleted

  • Nitisinone (NTBC) In Different Age Groups Of Patients With Alkaptonuria

    NCT01390077 · start 2011-01

    Registered record
    Registry ID
    NCT01390077
    Conditions
    Alkaptonuria
    Interventions
    Nitisinone
    Study type
    INTERVENTIONAL
    Phase
    Phase 2 / Phase 3
    Registry status
    Completed
    Start
    2011-01
    Completion
    2016-07
    Enrolment
    8

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

    ConditionAlkaptonuria
    InterventionNitisinone
    PhasePhase 2 / Phase 3
    Registry statusCompleted