Skip to content
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.

  • Long Term Nitric Oxide Bioavailability on Vascular Health in Chronic Obstructive Pulmonary Disease

    NCT02774226 · start 2015-09

    Registered record
    Registry ID
    NCT02774226
    Conditions
    Chronic Obstructive Pulmonary Disease (COPD)
    Interventions
    Tetrahydrobiopterin (BH4); Antioxidant Cocktail
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2015-09
    Completion
    2018-07-30
    Enrolment
    15

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

    ConditionChronic Obstructive Pulmonary Disease (COPD)
    InterventionTetrahydrobiopterin (BH4); Antioxidant Cocktail
    PhasePhase 2
    Registry statusCompleted

  • The Brain and Neuropsychological Functioning in Adults With Sapropterin Dihydrochloride Treated Phenylketonuria

    NCT02297347 · start 2015-04

    Registered record
    Registry ID
    NCT02297347
    Conditions
    Phenylketonuria
    Interventions
    Not recorded
    Study type
    OBSERVATIONAL
    Phase
    Not recorded
    Registry status
    Completed
    Start
    2015-04
    Completion
    2017-04
    Enrolment
    10

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

    ConditionPhenylketonuria
    InterventionNot recorded
    PhaseNot recorded
    Registry statusCompleted

  • Biological Variation of Phenylalanine in Patients With Hyperphenylalaninemia

    NCT01869972 · start 2013-05

    Registered record
    Registry ID
    NCT01869972
    Conditions
    Phenylketonuria; Hyperphenylalaninemia
    Interventions
    Not recorded
    Study type
    OBSERVATIONAL
    Phase
    Not recorded
    Registry status
    Completed
    Start
    2013-05
    Completion
    2015-12
    Enrolment
    32

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

    ConditionPhenylketonuria; Hyperphenylalaninemia
    InterventionNot recorded
    PhaseNot recorded
    Registry statusCompleted

  • A Pilot Study on Diurnal Variation

    NCT01806051 · start 2013-03

    Registered record
    Registry ID
    NCT01806051
    Conditions
    Phenylketonuria (PKU)
    Interventions
    Kuvan
    Study type
    INTERVENTIONAL
    Phase
    Not applicable
    Registry status
    Terminated
    Start
    2013-03
    Completion
    2014-05
    Enrolment
    6

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

    ConditionPhenylketonuria (PKU)
    InterventionKuvan
    PhaseNot applicable
    Registry statusTerminated

  • Phase 3 Open-label Study to Evaluate the Response and Safety of Kuvan® in Subjects With Phenylketonuria

    NCT01732471 · start 2012-11

    Registered record
    Registry ID
    NCT01732471
    Conditions
    Phenylketonuria
    Interventions
    Kuvan®
    Study type
    INTERVENTIONAL
    Phase
    Phase 3
    Registry status
    Completed
    Start
    2012-11
    Completion
    2013-10
    Enrolment
    90

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

    ConditionPhenylketonuria
    InterventionKuvan®
    PhasePhase 3
    Registry statusCompleted

  • Kuvan in People With Schizophrenia and Schizoaffective Disorder

    NCT01706965 · start 2012-10

    Registered record
    Registry ID
    NCT01706965
    Conditions
    Schizophrenia; Schizoaffective Disorder
    Interventions
    Kuvan; Multivitamin
    Study type
    INTERVENTIONAL
    Phase
    Phase 1 / Phase 2
    Registry status
    Completed
    Start
    2012-10
    Completion
    2016-06
    Enrolment
    27

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

    ConditionSchizophrenia; Schizoaffective Disorder
    InterventionKuvan; Multivitamin
    PhasePhase 1 / Phase 2
    Registry statusCompleted

  • The Effects of Sapropterin Dihydrochloride Supplementation on in Vivo Redox Status in Patients With Classical PKU

    NCT01650909 · start 2012-07

    Registered record
    Registry ID
    NCT01650909
    Conditions
    Classical Phenylketonuria(PKU)
    Interventions
    Not recorded
    Study type
    OBSERVATIONAL
    Phase
    Not recorded
    Registry status
    Withdrawn
    Start
    2012-07
    Completion
    Not recorded
    Enrolment
    0

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

    ConditionClassical Phenylketonuria(PKU)
    InterventionNot recorded
    PhaseNot recorded
    Registry statusWithdrawn

  • Effects of Kuvan on Melatonin Secretion

    NCT01617070 · start 2012-05

    Registered record
    Registry ID
    NCT01617070
    Conditions
    Phenylketonuria (PKU)
    Interventions
    Kuvan; Large Neutral Amino Acid Therapy
    Study type
    INTERVENTIONAL
    Phase
    Phase 4
    Registry status
    Completed
    Start
    2012-05
    Completion
    2014-12
    Enrolment
    10

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

    ConditionPhenylketonuria (PKU)
    InterventionKuvan; Large Neutral Amino Acid Therapy
    PhasePhase 4
    Registry statusCompleted

  • Randomized Controlled Trial to Assess the Effects of Sapropterin on Hepatic and Systemic Hemodynamics in Patients With Liver Cirrhosis and Portal Hypertension

    NCT01456286 · start 2011-10

    Registered record
    Registry ID
    NCT01456286
    Conditions
    Liver Cirrhosis; Portal Hypertension
    Interventions
    sapropterin
    Study type
    INTERVENTIONAL
    Phase
    Phase 2 / Phase 3
    Registry status
    Completed
    Start
    2011-10
    Completion
    2013-10
    Enrolment
    42

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

    ConditionLiver Cirrhosis; Portal Hypertension
    Interventionsapropterin
    PhasePhase 2 / Phase 3
    Registry statusCompleted

  • Study of Kuvan Treatment in Adults With GTPCH Deficiency

    NCT01425528 · start 2011-08

    Registered record
    Registry ID
    NCT01425528
    Conditions
    GTP Cyclohydrolase Deficiency
    Interventions
    Sapropterin
    Study type
    INTERVENTIONAL
    Phase
    Phase 1 / Phase 2
    Registry status
    Completed
    Start
    2011-08
    Completion
    2013-08
    Enrolment
    12

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

    ConditionGTP Cyclohydrolase Deficiency
    InterventionSapropterin
    PhasePhase 1 / Phase 2
    Registry statusCompleted

  • Cystic Fibrosis and Endothelial Function: At Rest and During Exercise

    NCT01772758 · start 2011-08

    Registered record
    Registry ID
    NCT01772758
    Conditions
    Cystic Fibrosis
    Interventions
    BH4 5mg; BH4 20mg; Antioxidant Cocktail
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2011-08
    Completion
    2016-06-21
    Enrolment
    64

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

    ConditionCystic Fibrosis
    InterventionBH4 5mg; BH4 20mg; Antioxidant Cocktail
    PhasePhase 2
    Registry statusCompleted

  • Phenylketonuria, Oxidative Stress, and BH4

    NCT01395394 · start 2011-06

    Registered record
    Registry ID
    NCT01395394
    Conditions
    Phenylketonuria
    Interventions
    Kuvan; Meal Challenge
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Terminated
    Start
    2011-06
    Completion
    2013-06
    Enrolment
    12

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

    ConditionPhenylketonuria
    InterventionKuvan; Meal Challenge
    PhasePhase 2
    Registry statusTerminated

  • Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy

    NCT01541397 · start 2011-06

    Registered record
    Registry ID
    NCT01541397
    Conditions
    Hyperphenylalaninemia; Phenylketonuria
    Interventions
    Sapropterin
    Study type
    INTERVENTIONAL
    Phase
    Not applicable
    Registry status
    Terminated
    Start
    2011-06
    Completion
    2012-08
    Enrolment
    6

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

    ConditionHyperphenylalaninemia; Phenylketonuria
    InterventionSapropterin
    PhaseNot applicable
    Registry statusTerminated

  • Exercise Intolerance in Renal Failure

    NCT01356966 · start 2011-05

    Registered record
    Registry ID
    NCT01356966
    Conditions
    Kidney Failure, Chronic
    Interventions
    Tetrahydrobiopterin; Placebo; Folate
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Completed
    Start
    2011-05
    Completion
    2014-05
    Enrolment
    74

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

    ConditionKidney Failure, Chronic
    InterventionTetrahydrobiopterin; Placebo; Folate
    PhasePhase 2
    Registry statusCompleted

  • Neuroimaging and Neurocognitive Assessment and Response to Sapropterin Dihydrochloride Treatment in Phenylketonuria

    NCT01412437 · start 2011-04

    Registered record
    Registry ID
    NCT01412437
    Conditions
    PKU
    Interventions
    diet; sapropterin dihydrochloride; sapropterin dihydrochloride
    Study type
    INTERVENTIONAL
    Phase
    Not applicable
    Registry status
    Withdrawn
    Start
    2011-04
    Completion
    2012-12
    Enrolment
    0

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

    ConditionPKU
    Interventiondiet; sapropterin dihydrochloride; sapropterin dihydrochloride
    PhaseNot applicable
    Registry statusWithdrawn