Skip to content
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 Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy

    NCT05115110 · start 2022-06-02

    Registered record
    Registry ID
    NCT05115110
    Conditions
    Spinal Muscular Atrophy (SMA)
    Interventions
    RO7204239; Placebo; Risdiplam
    Study type
    INTERVENTIONAL
    Phase
    Phase 2 / Phase 3
    Registry status
    Active not recruiting
    Start
    2022-06-02
    Completion
    2026-10-28
    Enrolment
    97

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

    ConditionSpinal Muscular Atrophy (SMA)
    InterventionRO7204239; Placebo; Risdiplam
    PhasePhase 2 / Phase 3
    Registry statusActive not recruiting

  • Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam

    NCT05156320 · start 2022-04-14

    Registered record
    Registry ID
    NCT05156320
    Conditions
    Spinal Muscular Atrophy; Spinal Muscular Atrophy Type 3; Spinal Muscular Atrophy Type 2; SMA; Neuromuscular Diseases; Muscular Atrophy; Atrophy; Muscular Atrophy, Spinal; Neuromuscular Manifestations; Anti-myostatin
    Interventions
    Apitegromab; Placebo
    Study type
    INTERVENTIONAL
    Phase
    Phase 3
    Registry status
    Completed
    Start
    2022-04-14
    Completion
    2024-12-18
    Enrolment
    188

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

    ConditionSpinal Muscular Atrophy; Spinal Muscular Atrophy Type 3; Spinal Muscular Atrophy Type 2; SMA +6 more
    InterventionApitegromab; Placebo
    PhasePhase 3
    Registry statusCompleted

  • Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

    NCT05232929 · start 2022-03-29

    Registered record
    Registry ID
    NCT05232929
    Conditions
    Spinal Muscular Atrophy
    Interventions
    Risdiplam
    Study type
    INTERVENTIONAL
    Phase
    Phase 4
    Registry status
    Active not recruiting
    Start
    2022-03-29
    Completion
    2026-10-02
    Enrolment
    403

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

    ConditionSpinal Muscular Atrophy
    InterventionRisdiplam
    PhasePhase 4
    Registry statusActive not recruiting

  • A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND)

    NCT05067790 · start 2022-01-21

    Registered record
    Registry ID
    NCT05067790
    Conditions
    Spinal Muscular Atrophy
    Interventions
    Nusinersen
    Study type
    INTERVENTIONAL
    Phase
    Phase 3
    Registry status
    Active not recruiting
    Start
    2022-01-21
    Completion
    2027-09-24
    Enrolment
    45

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

    ConditionSpinal Muscular Atrophy
    InterventionNusinersen
    PhasePhase 3
    Registry statusActive not recruiting

  • Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration

    NCT05219487 · start 2021-10-13

    Registered record
    Registry ID
    NCT05219487
    Conditions
    Spinal Muscular Atrophy
    Interventions
    Risdiplam
    Study type
    OBSERVATIONAL
    Phase
    Not recorded
    Registry status
    Completed
    Start
    2021-10-13
    Completion
    2025-12-31
    Enrolment
    18

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

    ConditionSpinal Muscular Atrophy
    InterventionRisdiplam
    PhaseNot recorded
    Registry statusCompleted

  • Bioavailability and Bioequivalence of Two Risdiplam Tablets in Healthy Participants

    NCT04718181 · start 2021-02-01

    Registered record
    Registry ID
    NCT04718181
    Conditions
    Muscular Atrophy, Spinal
    Interventions
    risdiplam; omeprazole
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2021-02-01
    Completion
    2023-01-28
    Enrolment
    131

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

    ConditionMuscular Atrophy, Spinal
    Interventionrisdiplam; omeprazole
    PhasePhase 1
    Registry statusCompleted

  • A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

    NCT03779334 · start 2019-08-07

    Registered record
    Registry ID
    NCT03779334
    Conditions
    Muscular Atrophy, Spinal
    Interventions
    Risdiplam
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Active not recruiting
    Start
    2019-08-07
    Completion
    2027-02-28
    Enrolment
    26

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

    ConditionMuscular Atrophy, Spinal
    InterventionRisdiplam
    PhasePhase 2
    Registry statusActive not recruiting

  • A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy Participants

    NCT03988907 · start 2019-06-18

    Registered record
    Registry ID
    NCT03988907
    Conditions
    Spinal Muscular Atrophy
    Interventions
    Risdiplam; Midazolam
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2019-06-18
    Completion
    2019-09-29
    Enrolment
    35

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

    ConditionSpinal Muscular Atrophy
    InterventionRisdiplam; Midazolam
    PhasePhase 1
    Registry statusCompleted

  • A Study to Investigate the Effect of Hepatic Impairment on the Pharmacokinetics and Safety and Tolerability of a Single Oral Dose of Risdiplam Compared to Matched Healthy Participants With Normal Hepatic Function

    NCT03920865 · start 2019-05-16

    Registered record
    Registry ID
    NCT03920865
    Conditions
    Muscular Atrophy, Spinal
    Interventions
    Risdiplam
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2019-05-16
    Completion
    2020-01-02
    Enrolment
    26

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

    ConditionMuscular Atrophy, Spinal
    InterventionRisdiplam
    PhasePhase 1
    Registry statusCompleted

  • Holter of Movement in Patients with SMA Undergoing Treatment.

    NCT04888702 · start 2018-08-08

    Registered record
    Registry ID
    NCT04888702
    Conditions
    Spinal Muscular Atrophy
    Interventions
    Actimyo
    Study type
    INTERVENTIONAL
    Phase
    Not applicable
    Registry status
    Unknown
    Start
    2018-08-08
    Completion
    2025-05-30
    Enrolment
    30

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

    ConditionSpinal Muscular Atrophy
    InterventionActimyo
    PhaseNot applicable
    Registry statusUnknown

  • A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) in Healthy Japanese Participants

    NCT03040635 · start 2017-03-22

    Registered record
    Registry ID
    NCT03040635
    Conditions
    Healthy Volunteers
    Interventions
    Risdiplam; Placebo
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2017-03-22
    Completion
    2017-10-02
    Enrolment
    24

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

    ConditionHealthy Volunteers
    InterventionRisdiplam; Placebo
    PhasePhase 1
    Registry statusCompleted

  • A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy

    NCT03032172 · start 2017-03-03

    Registered record
    Registry ID
    NCT03032172
    Conditions
    Spinal Muscular Atrophy
    Interventions
    Risdiplam
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2017-03-03
    Completion
    2025-02-07
    Enrolment
    174

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

    ConditionSpinal Muscular Atrophy
    InterventionRisdiplam
    PhasePhase 2
    Registry statusCompleted

  • A Study Investigating the Excretion Balance, Pharmacokinetics and Metabolism of a Single Oral Dose of [14C]-Labeled Risdiplam (RO7034067) in Healthy Male Participants

    NCT03036501 · start 2017-01-03

    Registered record
    Registry ID
    NCT03036501
    Conditions
    Healthy
    Interventions
    [^14C]-Risdiplam
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2017-01-03
    Completion
    2017-02-28
    Enrolment
    6

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

    ConditionHealthy
    Intervention[^14C]-Risdiplam
    PhasePhase 1
    Registry statusCompleted

  • Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy

    NCT02913482 · start 2016-12-23

    Registered record
    Registry ID
    NCT02913482
    Conditions
    Muscular Atrophy, Spinal
    Interventions
    Risdiplam
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2016-12-23
    Completion
    2023-12-22
    Enrolment
    62

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

    ConditionMuscular Atrophy, Spinal
    InterventionRisdiplam
    PhasePhase 2
    Registry statusCompleted

  • A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants

    NCT02908685 · start 2016-10-19

    Registered record
    Registry ID
    NCT02908685
    Conditions
    Muscular Atrophy, Spinal
    Interventions
    Placebo; Risdiplam
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2016-10-19
    Completion
    2023-10-02
    Enrolment
    231

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

    ConditionMuscular Atrophy, Spinal
    InterventionPlacebo; Risdiplam
    PhasePhase 2
    Registry statusCompleted