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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.

  • Assessing Physical Function in Sickle Cell Patients Taking Voxelotor

    NCT06023199 · start 2023-10-23

    Registered record
    Registry ID
    NCT06023199
    Conditions
    Sickle Cell Disease
    Interventions
    Voxelotor
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2023-10-23
    Completion
    2026-03-05
    Enrolment
    6

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

    ConditionSickle Cell Disease
    InterventionVoxelotor
    PhasePhase 2
    Registry statusCompleted

  • Voxelotor Brain Oxygenation and Neurocognitive Study

    NCT05228821 · start 2023-08-09

    Registered record
    Registry ID
    NCT05228821
    Conditions
    Sickle Cell Disease
    Interventions
    Voxelotor Oral Tablet
    Study type
    INTERVENTIONAL
    Phase
    Phase 4
    Registry status
    Withdrawn
    Start
    2023-08-09
    Completion
    2023-08-23
    Enrolment
    0

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

    ConditionSickle Cell Disease
    InterventionVoxelotor Oral Tablet
    PhasePhase 4
    Registry statusWithdrawn

  • Voxelotor CYP and Transporter Cocktail Interaction Study

    NCT05981365 · start 2023-04-17

    Registered record
    Registry ID
    NCT05981365
    Conditions
    Sickle Cell Disease
    Interventions
    Voxelotor; Bupropion; Repaglinide; Flurbiprofen; Omeprazole; Midazolam; Metformin; Furosemide; Rosuvastatin
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2023-04-17
    Completion
    2023-10-04
    Enrolment
    44

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

    ConditionSickle Cell Disease
    InterventionVoxelotor; Bupropion; Repaglinide; Flurbiprofen +5 more
    PhasePhase 1
    Registry statusCompleted

  • Hemolysis Related Complications in SCD. A Phase II Study With Voxelotor

    NCT05199766 · start 2023-03-22

    Registered record
    Registry ID
    NCT05199766
    Conditions
    Sickle Cell Disease
    Interventions
    Voxelotor 1500 mg oral per day (GBT440) for 48 weeks in patients with sickle-cell disease
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Unknown
    Start
    2023-03-22
    Completion
    2025-03-22
    Enrolment
    30

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

    ConditionSickle Cell Disease
    InterventionVoxelotor 1500 mg oral per day (GBT440) for 48 weeks in patients with sickle-cell disease
    PhasePhase 2
    Registry statusUnknown

  • Voxelotor Neurocognitive Function Study

    NCT05228834 · start 2022-06-24

    Registered record
    Registry ID
    NCT05228834
    Conditions
    Sickle Cell Disease
    Interventions
    Voxelotor Only Product in Oral Dose Form; Placebo
    Study type
    INTERVENTIONAL
    Phase
    Phase 3
    Registry status
    Terminated
    Start
    2022-06-24
    Completion
    2022-09-30
    Enrolment
    1

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

    ConditionSickle Cell Disease
    InterventionVoxelotor Only Product in Oral Dose Form; Placebo
    PhasePhase 3
    Registry statusTerminated

  • Resolution of Sickle Cell Leg Ulcers With Voxelotor

    NCT05561140 · start 2022-05-30

    Registered record
    Registry ID
    NCT05561140
    Conditions
    Sickle Cell Disease; Leg Ulcers
    Interventions
    Voxelotor Oral Tablet; Placebo
    Study type
    INTERVENTIONAL
    Phase
    Phase 3
    Registry status
    Terminated
    Start
    2022-05-30
    Completion
    2024-10-22
    Enrolment
    88

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

    ConditionSickle Cell Disease; Leg Ulcers
    InterventionVoxelotor Oral Tablet; Placebo
    PhasePhase 3
    Registry statusTerminated

  • Voxelotor for Improving Oxygen Saturation in Adults

    NCT05289570 · start 2022-05-03

    Registered record
    Registry ID
    NCT05289570
    Conditions
    Acute Lung Injury; End Stage Lung Disease
    Interventions
    Voxelotor
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Terminated
    Start
    2022-05-03
    Completion
    2023-07-04
    Enrolment
    3

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

    ConditionAcute Lung Injury; End Stage Lung Disease
    InterventionVoxelotor
    PhasePhase 2
    Registry statusTerminated

  • The Effect of Voxelotor on Cerebral Hemodynamic Response in Children With Sickle Cell Anemia

    NCT05018728 · start 2022-03-28

    Registered record
    Registry ID
    NCT05018728
    Conditions
    Sickle Cell Anemia in Children
    Interventions
    Voxelotor
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Terminated
    Start
    2022-03-28
    Completion
    2024-10-22
    Enrolment
    22

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

    ConditionSickle Cell Anemia in Children
    InterventionVoxelotor
    PhasePhase 2
    Registry statusTerminated

  • Oxbryta® Product Registry An Observational Study Designed to Evaluate the Effect of Oxbryta in Individuals With SCD

    NCT04930445 · start 2022-02-04

    Registered record
    Registry ID
    NCT04930445
    Conditions
    Sickle Cell Disease
    Interventions
    Oxbryta® (voxelotor) 500mg Tablets
    Study type
    OBSERVATIONAL
    Phase
    Not recorded
    Registry status
    Terminated
    Start
    2022-02-04
    Completion
    2024-10-10
    Enrolment
    265

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

    ConditionSickle Cell Disease
    InterventionOxbryta® (voxelotor) 500mg Tablets
    PhaseNot recorded
    Registry statusTerminated

  • Retrospective Real World Oxbryta® Data Collection and Analysis Study

    NCT04930328 · start 2021-03-20

    Registered record
    Registry ID
    NCT04930328
    Conditions
    Sickle Cell Disease
    Interventions
    Oxbryta® (voxelotor) 500-mg Tablets
    Study type
    OBSERVATIONAL
    Phase
    Not recorded
    Registry status
    Completed
    Start
    2021-03-20
    Completion
    2022-02-25
    Enrolment
    216

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

    ConditionSickle Cell Disease
    InterventionOxbryta® (voxelotor) 500-mg Tablets
    PhaseNot recorded
    Registry statusCompleted

  • A Voxelotor for Sickle Cell Anemia Patients at Highest Risk for Progression of Chronic Kidney Disease

    NCT04335721 · start 2021-03-16

    Registered record
    Registry ID
    NCT04335721
    Conditions
    Sickle Cell Disease; Sickle Cell Nephropathy
    Interventions
    Voxelotor
    Study type
    INTERVENTIONAL
    Phase
    Phase 1 / Phase 2
    Registry status
    Terminated
    Start
    2021-03-16
    Completion
    2024-10-07
    Enrolment
    12

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

    ConditionSickle Cell Disease; Sickle Cell Nephropathy
    InterventionVoxelotor
    PhasePhase 1 / Phase 2
    Registry statusTerminated

  • Actigraphy Improvement With Voxelotor (ActIVe) Study

    NCT04400487 · start 2020-12-21

    Registered record
    Registry ID
    NCT04400487
    Conditions
    Sickle Cell Disease; Sickle Cell Anemia
    Interventions
    Voxelotor
    Study type
    INTERVENTIONAL
    Phase
    Phase 4
    Registry status
    Completed
    Start
    2020-12-21
    Completion
    2022-09-13
    Enrolment
    25

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

    ConditionSickle Cell Disease; Sickle Cell Anemia
    InterventionVoxelotor
    PhasePhase 4
    Registry statusCompleted

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

    NCT04218084 · start 2020-11-11

    Registered record
    Registry ID
    NCT04218084
    Conditions
    Sickle Cell Disease
    Interventions
    Voxelotor; Placebo
    Study type
    INTERVENTIONAL
    Phase
    Phase 3
    Registry status
    Terminated
    Start
    2020-11-11
    Completion
    2024-11-06
    Enrolment
    236

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

    ConditionSickle Cell Disease
    InterventionVoxelotor; Placebo
    PhasePhase 3
    Registry statusTerminated

  • Voxelotor Sickle Cell Exercise Study

    NCT04581356 · start 2020-09-21

    Registered record
    Registry ID
    NCT04581356
    Conditions
    Sickle Cell Anemia
    Interventions
    Voxelotor
    Study type
    INTERVENTIONAL
    Phase
    Phase 4
    Registry status
    Completed
    Start
    2020-09-21
    Completion
    2021-12-29
    Enrolment
    14

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

    ConditionSickle Cell Anemia
    InterventionVoxelotor
    PhasePhase 4
    Registry statusCompleted

  • Dose Escalation Study to Evaluate the Safety, Tolerability, PK and PD of Voxelotor in Patients With SCD

    NCT04247594 · start 2020-01-09

    Registered record
    Registry ID
    NCT04247594
    Conditions
    Sickle Cell Disease
    Interventions
    Voxelotor
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Terminated
    Start
    2020-01-09
    Completion
    2021-06-08
    Enrolment
    6

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

    ConditionSickle Cell Disease
    InterventionVoxelotor
    PhasePhase 2
    Registry statusTerminated