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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 M. tuberculosis, MRSA and E. coli. 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 Registry of Cases of Spinal Muscular Atrophy

    NCT07805278 · start 2026-09

    Registered record
    Registry ID
    NCT07805278
    Conditions
    Spinal Muscular Atrophy
    Interventions
    Not recorded
    Study type
    OBSERVATIONAL
    Phase
    Not recorded
    Registry status
    Recruiting
    Start
    2026-09
    Completion
    2033-08
    Enrolment
    30

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

    ConditionSpinal Muscular Atrophy
    InterventionNot recorded
    PhaseNot recorded
    Registry statusRecruiting

  • A Study of Risdiplam in Participants With Type I and Type II Spinal Muscle Atrophy (SMA)

    NCT07531719 · start 2026-07-22

    Registered record
    Registry ID
    NCT07531719
    Conditions
    Spinal Muscle Atrophy
    Interventions
    Not recorded
    Study type
    OBSERVATIONAL
    Phase
    Not recorded
    Registry status
    Recruiting
    Start
    2026-07-22
    Completion
    2029-03-01
    Enrolment
    30

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

    ConditionSpinal Muscle Atrophy
    InterventionNot recorded
    PhaseNot recorded
    Registry statusRecruiting

  • A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 Years With Spinal Muscular Atrophy (SMA) Who Are Either New to SMA Treatment or Were Previously Treated With Risdiplam

    NCT07444476 · start 2026-04-03

    Registered record
    Registry ID
    NCT07444476
    Conditions
    Spinal Muscular Atrophy
    Interventions
    Salanersen
    Study type
    INTERVENTIONAL
    Phase
    Phase 3
    Registry status
    Recruiting
    Start
    2026-04-03
    Completion
    2032-06-22
    Enrolment
    90

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

    ConditionSpinal Muscular Atrophy
    InterventionSalanersen
    PhasePhase 3
    Registry statusRecruiting

  • Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1

    NCT07070999 · start 2026-01-06

    Registered record
    Registry ID
    NCT07070999
    Conditions
    Spinal Muscular Atrophy Type I
    Interventions
    GB221
    Study type
    INTERVENTIONAL
    Phase
    Phase 1 / Phase 2
    Registry status
    Recruiting
    Start
    2026-01-06
    Completion
    2029-04
    Enrolment
    22

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

    ConditionSpinal Muscular Atrophy Type I
    InterventionGB221
    PhasePhase 1 / Phase 2
    Registry statusRecruiting

  • A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy

    NCT07047144 · start 2025-09-15

    Registered record
    Registry ID
    NCT07047144
    Conditions
    Spinal Muscular Atrophy; SMA; Spinal Muscular Atrophy Type 2; Spinal Muscular Atrophy Type 3; Neuromuscular Manifestations; Anti-myostatin
    Interventions
    Apitegromab; Nusinersen; Risdiplam
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Recruiting
    Start
    2025-09-15
    Completion
    2029-03
    Enrolment
    52

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

    ConditionSpinal Muscular Atrophy; SMA; Spinal Muscular Atrophy Type 2; Spinal Muscular Atrophy Type 3 +2 more
    InterventionApitegromab; Nusinersen; Risdiplam
    PhasePhase 2
    Registry statusRecruiting

  • A Study of Dosing Patterns and Costs in Patients With Spinal Muscular Atrophy Receiving Disease Modifying Therapies

    NCT07378943 · start 2024-11-06

    Registered record
    Registry ID
    NCT07378943
    Conditions
    Muscular Atrophy, Spinal
    Interventions
    Not recorded
    Study type
    OBSERVATIONAL
    Phase
    Not recorded
    Registry status
    Completed
    Start
    2024-11-06
    Completion
    2025-03-30
    Enrolment
    4,114

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

    ConditionMuscular Atrophy, Spinal
    InterventionNot recorded
    PhaseNot recorded
    Registry statusCompleted

  • A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy

    NCT05861999 · start 2024-08-14

    Registered record
    Registry ID
    NCT05861999
    Conditions
    Muscular Atrophy, Spinal
    Interventions
    Risdiplam
    Study type
    INTERVENTIONAL
    Phase
    Phase 4
    Registry status
    Recruiting
    Start
    2024-08-14
    Completion
    2029-03-31
    Enrolment
    28

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

    ConditionMuscular Atrophy, Spinal
    InterventionRisdiplam
    PhasePhase 4
    Registry statusRecruiting

  • Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy

    NCT06321965 · start 2024-07-24

    Registered record
    Registry ID
    NCT06321965
    Conditions
    Spinal Muscular Atrophy
    Interventions
    evaluation of muscle function; First-line cognitive assessment; second-line cognitive assessment; Cardiac evaluation; MRI; Assessment of activity and muscle fatigue; Assessment of bulbar function; Evaluation of body composition and metabolism; Questionnaires; Biocollection; Skinfold measurement
    Study type
    INTERVENTIONAL
    Phase
    Not applicable
    Registry status
    Recruiting
    Start
    2024-07-24
    Completion
    2029-01-24
    Enrolment
    60

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

    ConditionSpinal Muscular Atrophy
    Interventionevaluation of muscle function; First-line cognitive assessment; second-line cognitive assessment; Cardiac evaluation +7 more
    PhaseNot applicable
    Registry statusRecruiting

  • A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy

    NCT05861986 · start 2024-05-30

    Registered record
    Registry ID
    NCT05861986
    Conditions
    Muscular Atrophy, Spinal
    Interventions
    Risdiplam
    Study type
    INTERVENTIONAL
    Phase
    Phase 4
    Registry status
    Recruiting
    Start
    2024-05-30
    Completion
    2029-03-31
    Enrolment
    28

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

    ConditionMuscular Atrophy, Spinal
    InterventionRisdiplam
    PhasePhase 4
    Registry statusRecruiting

  • A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

    NCT05808764 · start 2024-04-26

    Registered record
    Registry ID
    NCT05808764
    Conditions
    Muscular Atrophy, Spinal
    Interventions
    Risdiplam
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2024-04-26
    Completion
    2026-05-04
    Enrolment
    11

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

    ConditionMuscular Atrophy, Spinal
    InterventionRisdiplam
    PhasePhase 2
    Registry statusCompleted

  • MAP THE SMA: a Machine-learning Based Algorithm to Predict THErapeutic Response in Spinal Muscular Atrophy

    NCT05769465 · start 2023-04-01

    Registered record
    Registry ID
    NCT05769465
    Conditions
    Spinal Muscular Atrophy
    Interventions
    disease modifying treatments
    Study type
    OBSERVATIONAL
    Phase
    Not recorded
    Registry status
    Unknown
    Start
    2023-04-01
    Completion
    2026-04-01
    Enrolment
    247

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

    ConditionSpinal Muscular Atrophy
    Interventiondisease modifying treatments
    PhaseNot recorded
    Registry statusUnknown

  • Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam

    NCT05386680 · start 2023-01-12

    Registered record
    Registry ID
    NCT05386680
    Conditions
    Spinal Muscular Atrophy
    Interventions
    OAV101
    Study type
    INTERVENTIONAL
    Phase
    Phase 3
    Registry status
    Completed
    Start
    2023-01-12
    Completion
    2024-11-29
    Enrolment
    27

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

    ConditionSpinal Muscular Atrophy
    InterventionOAV101
    PhasePhase 3
    Registry statusCompleted

  • Mechanisms and Treatment of Exercise Intolerance and Persistent Fatigue in Spinal Muscular Atrophy

    NCT05518773 · start 2022-12-15

    Registered record
    Registry ID
    NCT05518773
    Conditions
    Spinal Muscular Atrophy
    Interventions
    Observational
    Study type
    OBSERVATIONAL
    Phase
    Not recorded
    Registry status
    Completed
    Start
    2022-12-15
    Completion
    2025-11-14
    Enrolment
    34

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

    ConditionSpinal Muscular Atrophy
    InterventionObservational
    PhaseNot recorded
    Registry statusCompleted

  • Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen

    NCT05522361 · start 2022-11-06

    Registered record
    Registry ID
    NCT05522361
    Conditions
    Spinal Muscular Atrophy
    Interventions
    Risdiplam
    Study type
    INTERVENTIONAL
    Phase
    Phase 4
    Registry status
    Unknown
    Start
    2022-11-06
    Completion
    2026-06-15
    Enrolment
    10

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

    ConditionSpinal Muscular Atrophy
    InterventionRisdiplam
    PhasePhase 4
    Registry statusUnknown

  • Adult SMA Research and Clinical Hub

    NCT06978985 · start 2022-07-29

    Registered record
    Registry ID
    NCT06978985
    Conditions
    Spinal Muscular Atrophy (SMA)
    Interventions
    Risdiplam; Nusinersen Injectable Product
    Study type
    OBSERVATIONAL
    Phase
    Not recorded
    Registry status
    Recruiting
    Start
    2022-07-29
    Completion
    2027-04-30
    Enrolment
    600

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

    ConditionSpinal Muscular Atrophy (SMA)
    InterventionRisdiplam; Nusinersen Injectable Product
    PhaseNot recorded
    Registry statusRecruiting