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AMR Drug RepurposingSmart Screeningresearch prototype

Risdiplam

FDA-approved product (for solution, oral), marketed as Evrysdi.

Registered studies
33
Lab records
0
Pathogens at ≥40%
3 of 4

Chemical structure

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

Existing / approved use

Documented · FDA, WHO, ChEMBL

Approved for

  • Atrophy
  • Proximal spinal muscular atrophy
  • Spinal muscular atrophy

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

WHO therapeutic group
Other drugs for disorders of the musculo-skeletal system
Anti-infective classification
Not classified as an anti-infective.

Repurposing investigation

This medicine is being investigated here for AI-predicted antimicrobial activity against MRSA, E. coli and M. tuberculosis. It was surfaced computationally for further investigation; it is not an established treatment for these infections.

AI-predicted activity

AI prediction · nothing measured
  • MRSA54.8% AI-predicted activityAt or above the ≥40% mark this site uses to surface candidates.
  • E. coli42.7% AI-predicted activityAt or above the ≥40% mark this site uses to surface candidates.
  • K. pneumoniae35.0% AI-predicted activityBelow the ≥40% mark this site uses to surface candidates.
  • M. tuberculosis55.1% 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

33registered 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 Risdiplam in ChEMBL Registered studies below

Docking

Computational · nothing measured

Dihydrofolate reductase ↗ (E. coli)

-8.3 kcal/mol

0better fit →-12

KPC-2 carbapenemase ↗ (K. pneumoniae)

-8.1 kcal/mol

0better fit →-12

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

-9.6 kcal/mol

0better fit →-12

Dihydrofolate reductase ↗ (MRSA)

-7.6 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 Risdiplam 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 Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) Given by Mouth in Healthy Volunteers

    NCT02633709 · start 2016-01-07

    Registered record
    Registry ID
    NCT02633709
    Conditions
    Spinal Muscular Atrophy
    Interventions
    Itraconazole; Placebo; Risdiplam
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2016-01-07
    Completion
    2016-08-04
    Enrolment
    33

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

    ConditionSpinal Muscular Atrophy
    InterventionItraconazole; Placebo; Risdiplam
    PhasePhase 1
    Registry statusCompleted

  • Registry Study of Assistive Devices, Medicines and Healthcare Measures in ALS, SMA and Other Neurological Diseases.

    NCT05852418 · start 2015-10-30

    Registered record
    Registry ID
    NCT05852418
    Conditions
    Amyotrophic Lateral Sclerosis
    Interventions
    Not recorded
    Study type
    OBSERVATIONAL
    Phase
    Not recorded
    Registry status
    Recruiting
    Start
    2015-10-30
    Completion
    2029-06
    Enrolment
    25,000

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

    ConditionAmyotrophic Lateral Sclerosis
    InterventionNot recorded
    PhaseNot recorded
    Registry statusRecruiting

  • An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

    NCT04256265

    Registered record
    Registry ID
    NCT04256265
    Conditions
    Muscular Atrophy, Spinal
    Interventions
    Risdiplam
    Study type
    EXPANDED_ACCESS
    Phase
    Not recorded
    Registry status
    Approved for marketing
    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.

    ConditionMuscular Atrophy, Spinal
    InterventionRisdiplam
    PhaseNot recorded
    Registry statusApproved for marketing