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AMR Drug RepurposingSmart Screeningresearch prototype

Voxelotor

FDA-approved product (tablet, oral), marketed as Oxbryta.

Registered studies
31
Lab records
0
Pathogens at ≥40%
1 of 4

Chemical structure

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

Existing / approved use

Documented · FDA, WHO, ChEMBL

Approved for

  • Anemia
  • Anemia (phenotype)
  • Sickle cell anemia

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

WHO therapeutic group
Other hematological agents
FDA pharmacologic class
Hemoglobin S Polymerization 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
  • MRSA34.7% AI-predicted activityBelow the ≥40% mark this site uses to surface candidates.
  • E. coli27.8% AI-predicted activityBelow the ≥40% mark this site uses to surface candidates.
  • K. pneumoniae22.0% AI-predicted activityBelow the ≥40% mark this site uses to surface candidates.
  • M. tuberculosis56.8% 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

31registered 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 Voxelotor in ChEMBL Registered studies below

Docking

Computational · nothing measured

Dihydrofolate reductase ↗ (E. coli)

-7.3 kcal/mol

0better fit →-12

KPC-2 carbapenemase ↗ (K. pneumoniae)

-7.7 kcal/mol

0better fit →-12

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

-7.9 kcal/mol

0better fit →-12

Dihydrofolate reductase ↗ (MRSA)

-8.5 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 Voxelotor 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.

  • Open-Label Extension of Voxelotor

    NCT04188509 · start 2019-11-18

    Registered record
    Registry ID
    NCT04188509
    Conditions
    Sickle Cell Disease
    Interventions
    Voxelotor
    Study type
    INTERVENTIONAL
    Phase
    Phase 3
    Registry status
    Terminated
    Start
    2019-11-18
    Completion
    2024-11-01
    Enrolment
    162

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

    ConditionSickle Cell Disease
    InterventionVoxelotor
    PhasePhase 3
    Registry statusTerminated

  • Study to Assess the Effect of Long-term Treatment With Voxelotor in Participants Who Have Completed Treatment in Study GBT440-031

    NCT03573882 · start 2018-06-06

    Registered record
    Registry ID
    NCT03573882
    Conditions
    Sickle Cell Disease
    Interventions
    Voxelotor
    Study type
    INTERVENTIONAL
    Phase
    Phase 3
    Registry status
    Terminated
    Start
    2018-06-06
    Completion
    2024-11-12
    Enrolment
    179

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

    ConditionSickle Cell Disease
    InterventionVoxelotor
    PhasePhase 3
    Registry statusTerminated

  • Single-Dose PK and Safety Study of GBT440 in Subjects With Hepatic Impairment

    NCT03114540 · start 2017-03-17

    Registered record
    Registry ID
    NCT03114540
    Conditions
    Hepatic Impairment
    Interventions
    GBT440
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2017-03-17
    Completion
    2018-03-22
    Enrolment
    29

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

    ConditionHepatic Impairment
    InterventionGBT440
    PhasePhase 1
    Registry statusCompleted

  • Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE)

    NCT03036813 · start 2016-12

    Registered record
    Registry ID
    NCT03036813
    Conditions
    Sickle Cell Disease
    Interventions
    voxelotor; Placebo
    Study type
    INTERVENTIONAL
    Phase
    Phase 3
    Registry status
    Completed
    Start
    2016-12
    Completion
    2019-10-08
    Enrolment
    449

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

    ConditionSickle Cell Disease
    Interventionvoxelotor; Placebo
    PhasePhase 3
    Registry statusCompleted

  • Study to Evaluate Effects of Investigational Drug on Oxygenation in Healthy Volunteers Under Hypoxic Conditions

    NCT03051711 · start 2016-12

    Registered record
    Registry ID
    NCT03051711
    Conditions
    Cardio-pulmonary Function
    Interventions
    GBT440
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Terminated
    Start
    2016-12
    Completion
    2018-02-21
    Enrolment
    14

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

    ConditionCardio-pulmonary Function
    InterventionGBT440
    PhasePhase 1
    Registry statusTerminated

  • Study to Evaluate the Effect of GBT440 Administered to Subjects With IPF on Supplemental Oxygen at Rest

    NCT02989168 · start 2016-11

    Registered record
    Registry ID
    NCT02989168
    Conditions
    Idiopathic Pulmonary Fibrosis; Hypoxemia
    Interventions
    GBT440
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Terminated
    Start
    2016-11
    Completion
    2017-10-23
    Enrolment
    14

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

    ConditionIdiopathic Pulmonary Fibrosis; Hypoxemia
    InterventionGBT440
    PhasePhase 2
    Registry statusTerminated

  • Single-Dose PK Study of GBT440 in Subjects With Renal Impairment

    NCT03161015 · start 2016-11

    Registered record
    Registry ID
    NCT03161015
    Conditions
    Renal Impairment
    Interventions
    GBT440
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2016-11
    Completion
    2017-07-06
    Enrolment
    16

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

    ConditionRenal Impairment
    InterventionGBT440
    PhasePhase 1
    Registry statusCompleted

  • An Extension Study to Further Evaluate the Safety, Tolerability of GBT440 in Patients With Sickle Cell Disease Who Participated in the Study GBT440-001

    NCT03041909 · start 2016-08

    Registered record
    Registry ID
    NCT03041909
    Conditions
    Sickle Cell Disease
    Interventions
    GBT440
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2016-08
    Completion
    2017-08
    Enrolment
    5

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

    ConditionSickle Cell Disease
    InterventionGBT440
    PhasePhase 2
    Registry statusCompleted

  • Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease

    NCT02850406 · start 2016-07-05

    Registered record
    Registry ID
    NCT02850406
    Conditions
    Sickle Cell Disease
    Interventions
    Voxelotor
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Terminated
    Start
    2016-07-05
    Completion
    2023-10-02
    Enrolment
    147

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

    ConditionSickle Cell Disease
    InterventionVoxelotor
    PhasePhase 2
    Registry statusTerminated

  • Study to Evaluate the Safety and Tolerability of GBT440 Administered to Subjects With IPF

    NCT02846324 · start 2016-06

    Registered record
    Registry ID
    NCT02846324
    Conditions
    Idiopathic Pulmonary Fibrosis; Hypoxemia
    Interventions
    GBT440; Placebo
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2016-06
    Completion
    2017-11
    Enrolment
    39

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

    ConditionIdiopathic Pulmonary Fibrosis; Hypoxemia
    InterventionGBT440; Placebo
    PhasePhase 2
    Registry statusCompleted

  • Drug Interaction Study of GBT440 With Caffeine, S-warfarin, Omeprazole, and Midazolam in Healthy Subjects

    NCT02567682 · start 2015-09

    Registered record
    Registry ID
    NCT02567682
    Conditions
    Sickle Cell Disease
    Interventions
    GBT440
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2015-09
    Completion
    2016-05
    Enrolment
    24

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

    ConditionSickle Cell Disease
    InterventionGBT440
    PhasePhase 1
    Registry statusCompleted

  • A Single-Dose Relative Bioavailability Study Of GBT440 300 mg Capsules in Healthy Subjects

    NCT02567695 · start 2015-09

    Registered record
    Registry ID
    NCT02567695
    Conditions
    Sickle Cell Disease
    Interventions
    GBT440
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2015-09
    Completion
    2016-06
    Enrolment
    26

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

    ConditionSickle Cell Disease
    InterventionGBT440
    PhasePhase 1
    Registry statusCompleted

  • A Study of the Absorption, Metabolism, and Excretion of GBT440 in Healthy Male Subjects

    NCT02497924 · start 2015-06

    Registered record
    Registry ID
    NCT02497924
    Conditions
    Anemia, Sickle Cell
    Interventions
    GBT440
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2015-06
    Completion
    2016-07
    Enrolment
    7

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

    ConditionAnemia, Sickle Cell
    InterventionGBT440
    PhasePhase 1
    Registry statusCompleted

  • A Study of the Safety, Blood Levels and Biological Effects of GBT440 in Healthy Subjects and Subjects With Sickle Cell Disease

    NCT02285088 · start 2014-12

    Registered record
    Registry ID
    NCT02285088
    Conditions
    Healthy Subjects; Sickle Cell Disease
    Interventions
    GBT440; Placebo
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2014-12
    Completion
    2017-05
    Enrolment
    133

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

    ConditionHealthy Subjects; Sickle Cell Disease
    InterventionGBT440; Placebo
    PhasePhase 1
    Registry statusCompleted

  • Expanded Access to Voxelotor for Patients With Sickle Cell Disease Who Have No Alternative Treatment Options

    NCT03943615

    Registered record
    Registry ID
    NCT03943615
    Conditions
    Sickle Cell Disease
    Interventions
    Voxelotor
    Study type
    EXPANDED_ACCESS
    Phase
    Not recorded
    Registry status
    No longer available
    Start
    Not recorded
    Completion
    Not recorded
    Enrolment
    Not recorded

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

    ConditionSickle Cell Disease
    InterventionVoxelotor
    PhaseNot recorded
    Registry statusNo longer available