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AMR Drug RepurposingSmart Screeningresearch prototype

Sapropterin Dihydrochloride

FDA-approved product (tablet, oral), marketed as Kuvan, Sapropterin Dihydrochloride, Zelvysia.

Registered studies
50+
Lab records
0
Pathogens at ≥40%
2 of 4

Chemical structure

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

Existing / approved use

Documented · FDA, WHO, ChEMBL

Approved for

  • Hyperphenylalaninemia
  • Phenylketonuria

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

WHO therapeutic group
Various alimentary tract and metabolism products
Anti-infective classification
Not classified as an anti-infective.

Repurposing investigation

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

AI-predicted activity

AI prediction · nothing measured
  • MRSA48.0% AI-predicted activityAt or above the ≥40% mark this site uses to surface candidates.
  • E. coli40.4% AI-predicted activityAt or above the ≥40% mark this site uses to surface candidates.
  • K. pneumoniae35.2% AI-predicted activityBelow the ≥40% mark this site uses to surface candidates.
  • M. tuberculosis28.4% AI-predicted activityBelow 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

50+registered studies name this medicine, for any condition. The registry search keeps the first 50, so there may be more. See them below.

Experimental

No evidence found

No laboratory measurement against the four pathogens in the ChEMBL records loaded here.

Open Sapropterin Dihydrochloride in ChEMBL Registered studies below

Docking

Computational · nothing measured

Dihydrofolate reductase ↗ (E. coli)

-6.3 kcal/mol

0better fit →-12

KPC-2 carbapenemase ↗ (K. pneumoniae)

-7.8 kcal/mol

0better fit →-12

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

-7.2 kcal/mol

0better fit →-12

Dihydrofolate reductase ↗ (MRSA)

-6.8 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 Sapropterin Dihydrochloride 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 of the Effects of 6R-BH4 on Blood Pressure in Subjects With Poorly Controlled Systemic Hypertension

    NCT00325962 · start 2006-05

    Registered record
    Registry ID
    NCT00325962
    Conditions
    Hypertension
    Interventions
    6R-BH4 (sapropterin dihydrochloride)
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2006-05
    Completion
    2008-12
    Enrolment
    84

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

    ConditionHypertension
    Intervention6R-BH4 (sapropterin dihydrochloride)
    PhasePhase 2
    Registry statusCompleted

  • Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet

    NCT00272792 · start 2006-02

    Registered record
    Registry ID
    NCT00272792
    Conditions
    Phenylketonurias
    Interventions
    Sapropterin Dihydrochloride; Placebo
    Study type
    INTERVENTIONAL
    Phase
    Phase 3
    Registry status
    Completed
    Start
    2006-02
    Completion
    2006-11
    Enrolment
    45

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

    ConditionPhenylketonurias
    InterventionSapropterin Dihydrochloride; Placebo
    PhasePhase 3
    Registry statusCompleted

  • Response to Phenylketonuria to Tetrahydrobiopterin (BH4)

    NCT00244218 · start 2005-04

    Registered record
    Registry ID
    NCT00244218
    Conditions
    Phenylketonuria
    Interventions
    tetrahydrobiopterin (BH4)
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Terminated
    Start
    2005-04
    Completion
    2015-01
    Enrolment
    57

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

    ConditionPhenylketonuria
    Interventiontetrahydrobiopterin (BH4)
    PhasePhase 1
    Registry statusTerminated

  • Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels

    NCT00104247 · start 2005-03

    Registered record
    Registry ID
    NCT00104247
    Conditions
    Phenylketonurias
    Interventions
    sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin
    Study type
    INTERVENTIONAL
    Phase
    Phase 3
    Registry status
    Completed
    Start
    2005-03
    Completion
    2006-02
    Enrolment
    89

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

    ConditionPhenylketonurias
    Interventionsapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin
    PhasePhase 3
    Registry statusCompleted

  • Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria

    NCT00104260 · start 2004-12

    Registered record
    Registry ID
    NCT00104260
    Conditions
    Phenylketonurias
    Interventions
    sapropterin dihydrochloride
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2004-12
    Completion
    2005-11
    Enrolment
    700

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

    ConditionPhenylketonurias
    Interventionsapropterin dihydrochloride
    PhasePhase 2
    Registry statusCompleted