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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 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
  • 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.

  • Evaluation of Behavior, Executive Function, Neurotransmitter Function and Genomic Expression Kuvan Nonresponders

    NCT01274026 · start 2011-01

    Registered record
    Registry ID
    NCT01274026
    Conditions
    Phenylketonuria; Behavior and Behavior Mechanisms; PAH Gene Expression
    Interventions
    sapropterin dihydrochloride
    Study type
    OBSERVATIONAL
    Phase
    Not recorded
    Registry status
    Completed
    Start
    2011-01
    Completion
    2013-12
    Enrolment
    21

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

    ConditionPhenylketonuria; Behavior and Behavior Mechanisms; PAH Gene Expression
    Interventionsapropterin dihydrochloride
    PhaseNot recorded
    Registry statusCompleted

  • Nitric Oxide Bioavailability in Chronic Obstructive Pulmonary Disease (COPD)

    NCT01398943 · start 2010-09

    Registered record
    Registry ID
    NCT01398943
    Conditions
    Pulmonary Disease, Chronic Obstructive
    Interventions
    Tetrahydrobiopterin (BH4); Antioxidant Cocktail
    Study type
    INTERVENTIONAL
    Phase
    Not applicable
    Registry status
    Completed
    Start
    2010-09
    Completion
    2015-06
    Enrolment
    60

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

    ConditionPulmonary Disease, Chronic Obstructive
    InterventionTetrahydrobiopterin (BH4); Antioxidant Cocktail
    PhaseNot applicable
    Registry statusCompleted

  • Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients

    NCT01114737 · start 2010-08

    Registered record
    Registry ID
    NCT01114737
    Conditions
    Phenylketonuria
    Interventions
    Sapropterin dihydrochloride; Placebo
    Study type
    INTERVENTIONAL
    Phase
    Phase 3
    Registry status
    Completed
    Start
    2010-08
    Completion
    2013-03
    Enrolment
    206

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

    ConditionPhenylketonuria
    InterventionSapropterin dihydrochloride; Placebo
    PhasePhase 3
    Registry statusCompleted

  • Tetrahydrobiopterin Treatment in Children With Idiopathic Cognitive Developmental Disorders

    NCT01141595 · start 2010-07

    Registered record
    Registry ID
    NCT01141595
    Conditions
    Autism Spectrum Disorder
    Interventions
    sapropterin dihydrochloride
    Study type
    INTERVENTIONAL
    Phase
    Not applicable
    Registry status
    Completed
    Start
    2010-07
    Completion
    2011-09
    Enrolment
    10

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

    ConditionAutism Spectrum Disorder
    Interventionsapropterin dihydrochloride
    PhaseNot applicable
    Registry statusCompleted

  • Response to Kuvan® in Subjects With Phenylketonuria (PKU) in a 4 Weeks Testing Period

    NCT01082328 · start 2010-05

    Registered record
    Registry ID
    NCT01082328
    Conditions
    Phenylketonuria
    Interventions
    Kuvan®
    Study type
    INTERVENTIONAL
    Phase
    Phase 4
    Registry status
    Completed
    Start
    2010-05
    Completion
    2012-05
    Enrolment
    59

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

    ConditionPhenylketonuria
    InterventionKuvan®
    PhasePhase 4
    Registry statusCompleted

  • Open-Label Extension Study of Kuvan for Autism

    NCT00943579 · start 2009-08

    Registered record
    Registry ID
    NCT00943579
    Conditions
    Autistic Disorder
    Interventions
    Kuvan®
    Study type
    INTERVENTIONAL
    Phase
    Phase 2 / Phase 3
    Registry status
    Completed
    Start
    2009-08
    Completion
    2012-03
    Enrolment
    41

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

    ConditionAutistic Disorder
    InterventionKuvan®
    PhasePhase 2 / Phase 3
    Registry statusCompleted

  • Behavioral Effects of Kuvan in Children With Mild Phenylketonuria

    NCT00827762 · start 2009-01

    Registered record
    Registry ID
    NCT00827762
    Conditions
    Phenylketonuria
    Interventions
    Kuvan
    Study type
    OBSERVATIONAL
    Phase
    Not recorded
    Registry status
    Terminated
    Start
    2009-01
    Completion
    2010-01
    Enrolment
    2

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

    ConditionPhenylketonuria
    InterventionKuvan
    PhaseNot recorded
    Registry statusTerminated

  • Oral 6R-BH4 for the Treatment of Isolated Systolic Hypertension and Endothelial Dysfunction

    NCT00802893 · start 2008-12

    Registered record
    Registry ID
    NCT00802893
    Conditions
    Isolated Systolic Hypertension; Endothelial Dysfunction
    Interventions
    6R-BH4; Placebo
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Terminated
    Start
    2008-12
    Completion
    2009-05
    Enrolment
    3

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

    ConditionIsolated Systolic Hypertension; Endothelial Dysfunction
    Intervention6R-BH4; Placebo
    PhasePhase 2
    Registry statusTerminated

  • Nutritional and Neurotransmitter Changes in PKU Subjects on BH4

    NCT00688844 · start 2008-10

    Registered record
    Registry ID
    NCT00688844
    Conditions
    Phenylketonuria
    Interventions
    KuvanTM Therapy
    Study type
    OBSERVATIONAL
    Phase
    Not recorded
    Registry status
    Completed
    Start
    2008-10
    Completion
    2010-10
    Enrolment
    58

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

    ConditionPhenylketonuria
    InterventionKuvanTM Therapy
    PhaseNot recorded
    Registry statusCompleted

  • A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects

    NCT00789568 · start 2008-10

    Registered record
    Registry ID
    NCT00789568
    Conditions
    Phenylketonuria
    Interventions
    sapropterin dihydrochloride; Moxifloxacin; Moxifloxacin placebo
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2008-10
    Completion
    2009-10
    Enrolment
    56

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

    ConditionPhenylketonuria
    Interventionsapropterin dihydrochloride; Moxifloxacin; Moxifloxacin placebo
    PhasePhase 1
    Registry statusCompleted

  • PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry

    NCT00778206 · start 2008-09

    Registered record
    Registry ID
    NCT00778206
    Conditions
    Phenylketonuria; Hyperphenylalaninaemia
    Interventions
    Kuvan
    Study type
    OBSERVATIONAL
    Phase
    Not recorded
    Registry status
    Completed
    Start
    2008-09
    Completion
    2021-01-29
    Enrolment
    1,887

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

    ConditionPhenylketonuria; Hyperphenylalaninaemia
    InterventionKuvan
    PhaseNot recorded
    Registry statusCompleted

  • Sapropterin in Individuals With Phenylketonuria

    NCT00730080 · start 2008-07

    Registered record
    Registry ID
    NCT00730080
    Conditions
    Phenylketonuria
    Interventions
    Sapropterin (Kuvan)
    Study type
    OBSERVATIONAL
    Phase
    Not recorded
    Registry status
    Completed
    Start
    2008-07
    Completion
    2018-05
    Enrolment
    45

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

    ConditionPhenylketonuria
    InterventionSapropterin (Kuvan)
    PhaseNot recorded
    Registry statusCompleted

  • Tetrahydrobiopterin in Patients With Chronic Kidney Disease (CKD) and Albuminuria

    NCT00625820 · start 2008-05

    Registered record
    Registry ID
    NCT00625820
    Conditions
    Kidney Disease; Albuminuria
    Interventions
    6R BH4; Vitamin C
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2008-05
    Completion
    2008-09
    Enrolment
    17

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

    ConditionKidney Disease; Albuminuria
    Intervention6R BH4; Vitamin C
    PhasePhase 2
    Registry statusCompleted

  • A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction

    NCT00532844 · start 2007-09

    Registered record
    Registry ID
    NCT00532844
    Conditions
    Endothelial Dysfunction
    Interventions
    Sapropterin Dihydrochloride; Sapropterin Dihydrochloride and Vitamin C
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2007-09
    Completion
    2009-03
    Enrolment
    52

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

    ConditionEndothelial Dysfunction
    InterventionSapropterin Dihydrochloride; Sapropterin Dihydrochloride and Vitamin C
    PhasePhase 2
    Registry statusCompleted

  • A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell Disease

    NCT00445978 · start 2007-05

    Registered record
    Registry ID
    NCT00445978
    Conditions
    Sickle Cell Disease
    Interventions
    Sapropterin Dihydrochloride
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2007-05
    Completion
    2009-06
    Enrolment
    32

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

    ConditionSickle Cell Disease
    InterventionSapropterin Dihydrochloride
    PhasePhase 2
    Registry statusCompleted