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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 Single Patient Study of Sapropterin for Multisystem Smooth Muscle Dysfunction Syndrome

    NCT07574034 · start 2026-06-01

    Registered record
    Registry ID
    NCT07574034
    Conditions
    Multisystemic Smooth Muscle Dysfunction Syndrome
    Interventions
    Kuvan (Sapropterine)
    Study type
    INTERVENTIONAL
    Phase
    Not applicable
    Registry status
    Not yet recruiting
    Start
    2026-06-01
    Completion
    2030-06-01
    Enrolment
    1

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

    ConditionMultisystemic Smooth Muscle Dysfunction Syndrome
    InterventionKuvan (Sapropterine)
    PhaseNot applicable
    Registry statusNot yet recruiting

  • Impact Of A Phe-Restricted Diet On Gut Health In Children With PKU

    NCT07728032 · start 2025-10-01

    Registered record
    Registry ID
    NCT07728032
    Conditions
    Phenylketonuria
    Interventions
    Phenylalanine-restricted diet alone; Sapropterin Dihydrochloride; Sepiapterin
    Study type
    OBSERVATIONAL
    Phase
    Not recorded
    Registry status
    Recruiting
    Start
    2025-10-01
    Completion
    2026-12
    Enrolment
    148

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

    ConditionPhenylketonuria
    InterventionPhenylalanine-restricted diet alone; Sapropterin Dihydrochloride; Sepiapterin
    PhaseNot recorded
    Registry statusRecruiting

  • Evaluation of BH4 Responsiveness in Our PKU Patients

    NCT07255599 · start 2025-09-01

    Registered record
    Registry ID
    NCT07255599
    Conditions
    Phenylketonuria (PKU)
    Interventions
    Sapropterin Dihydrochloride (BH4, tetrahydrobiopterin)
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Active not recruiting
    Start
    2025-09-01
    Completion
    2026-09-01
    Enrolment
    20

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

    ConditionPhenylketonuria (PKU)
    InterventionSapropterin Dihydrochloride (BH4, tetrahydrobiopterin)
    PhasePhase 1
    Registry statusActive not recruiting

  • Study on Sapropterin Dihydrochloride Oral Suspension in Healthy Subjects Under Fed Conditions

    NCT06987773 · start 2025-08-20

    Registered record
    Registry ID
    NCT06987773
    Conditions
    Bioequivalence
    Interventions
    RLF-OD032 100 mg/mL oral suspension; Kuvan 100 MG Powder for Oral Solution
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2025-08-20
    Completion
    2025-10-28
    Enrolment
    42

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

    ConditionBioequivalence
    InterventionRLF-OD032 100 mg/mL oral suspension; Kuvan 100 MG Powder for Oral Solution
    PhasePhase 1
    Registry statusCompleted

  • BH4 in the Prevention and Treatment of Radiation-induced Skin Reactions for Head and Neck Squamous Cell Carcinoma (Including Nasopharyngeal Carcinoma)

    NCT05299203 · start 2022-05

    Registered record
    Registry ID
    NCT05299203
    Conditions
    Head and Neck Squamous Cell Carcinoma
    Interventions
    Sapropterin dihydrochloride tablets
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Unknown
    Start
    2022-05
    Completion
    2023-05
    Enrolment
    18

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

    ConditionHead and Neck Squamous Cell Carcinoma
    InterventionSapropterin dihydrochloride tablets
    PhasePhase 1
    Registry statusUnknown

  • A Study for Assessing the Efficacy and Safety of BH4 in Radiation Enteritis

    NCT05138887 · start 2022-03-01

    Registered record
    Registry ID
    NCT05138887
    Conditions
    Radiation Enteritis
    Interventions
    Tetrahydrobiopterin
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Unknown
    Start
    2022-03-01
    Completion
    2024-03-01
    Enrolment
    366

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

    ConditionRadiation Enteritis
    InterventionTetrahydrobiopterin
    PhasePhase 2
    Registry statusUnknown

  • Novel Approaches for Improving Vascular Function in Veterans With HFpEF

    NCT06312748 · start 2021-05-19

    Registered record
    Registry ID
    NCT06312748
    Conditions
    Heart Failure With Preserved Ejection Fraction; Inflammation
    Interventions
    L-Citrulline; Placebo for L-Citrulline; BH4; Placebo for BH4; Atorvastatin; Placebo for Atorvastatin
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Recruiting
    Start
    2021-05-19
    Completion
    2030-09-30
    Enrolment
    90

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

    ConditionHeart Failure With Preserved Ejection Fraction; Inflammation
    InterventionL-Citrulline; Placebo for L-Citrulline; BH4; Placebo for BH4 +2 more
    PhasePhase 1
    Registry statusRecruiting

  • O2 Transport and Utilization in Health and Lung Disease

    NCT04014712 · start 2021-04

    Registered record
    Registry ID
    NCT04014712
    Conditions
    COPD; Tetrahydrobiopterin Deficiency; Oxidative Stress
    Interventions
    Tetrahydrobiopterin; Placebo oral tablet
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Withdrawn
    Start
    2021-04
    Completion
    2022-08
    Enrolment
    0

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

    ConditionCOPD; Tetrahydrobiopterin Deficiency; Oxidative Stress
    InterventionTetrahydrobiopterin; Placebo oral tablet
    PhasePhase 1
    Registry statusWithdrawn

  • L-arginine Study for Persistent Symptoms of Schizophrenia

    NCT04054973 · start 2019-09-11

    Registered record
    Registry ID
    NCT04054973
    Conditions
    Schizo Affective Disorder; Schizophrenia
    Interventions
    L-arginine; Sapropterin Dihydrochloride
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Withdrawn
    Start
    2019-09-11
    Completion
    2021-12-31
    Enrolment
    0

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

    ConditionSchizo Affective Disorder; Schizophrenia
    InterventionL-arginine; Sapropterin Dihydrochloride
    PhasePhase 2
    Registry statusWithdrawn

  • Nutritional Status in Phenylketonuria

    NCT03820804 · start 2019-01-07

    Registered record
    Registry ID
    NCT03820804
    Conditions
    Phenylketonurias
    Interventions
    Diet; Sapropterin
    Study type
    OBSERVATIONAL
    Phase
    Not recorded
    Registry status
    Completed
    Start
    2019-01-07
    Completion
    2019-09-10
    Enrolment
    94

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

    ConditionPhenylketonurias
    InterventionDiet; Sapropterin
    PhaseNot recorded
    Registry statusCompleted

  • The Effectiveness of Kuvan in Amish PKU Patients

    NCT02677870 · start 2018-01-22

    Registered record
    Registry ID
    NCT02677870
    Conditions
    Phenylketonuria
    Interventions
    saproterin dihydrochloride; Diet treatment
    Study type
    INTERVENTIONAL
    Phase
    Phase 4
    Registry status
    Completed
    Start
    2018-01-22
    Completion
    2019-01-01
    Enrolment
    7

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

    ConditionPhenylketonuria
    Interventionsaproterin dihydrochloride; Diet treatment
    PhasePhase 4
    Registry statusCompleted

  • The Effect of Antioxidants on Skin Blood Flow-BH4

    NCT03680573 · start 2018-01-08

    Registered record
    Registry ID
    NCT03680573
    Conditions
    Cardiovascular Diseases; Cardiovascular Risk Factor; Vasoconstriction
    Interventions
    Control: Lacated Ringers; Apocynin; Allopurinol; BH4; NG Nitro L Arginine Methyl Ester; Sodium Nitroprusside; Acetyl-ß-methylcholine chloride
    Study type
    INTERVENTIONAL
    Phase
    Phase 1
    Registry status
    Completed
    Start
    2018-01-08
    Completion
    2018-05-05
    Enrolment
    16

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

    ConditionCardiovascular Diseases; Cardiovascular Risk Factor; Vasoconstriction
    InterventionControl: Lacated Ringers; Apocynin; Allopurinol; BH4 +3 more
    PhasePhase 1
    Registry statusCompleted

  • Retrospective Observational Safety Effectiveness With Kuvan in hpA

    NCT03864029 · start 2017-10-10

    Registered record
    Registry ID
    NCT03864029
    Conditions
    Tetrahydrobiopterin Deficiency
    Interventions
    KUVAN
    Study type
    OBSERVATIONAL
    Phase
    Not recorded
    Registry status
    Completed
    Start
    2017-10-10
    Completion
    2018-07-25
    Enrolment
    26

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

    ConditionTetrahydrobiopterin Deficiency
    InterventionKUVAN
    PhaseNot recorded
    Registry statusCompleted

  • Neurovascular Transduction During Exercise in Chronic Kidney Disease

    NCT02947750 · start 2016-10

    Registered record
    Registry ID
    NCT02947750
    Conditions
    Renal Insufficiency, Chronic
    Interventions
    6R-BH4; 6R-BH4 placebo; Folic acid; Exercise training; Stretching; Histidine and beta-alanine supplementation; Histidine and beta-alanine placebo
    Study type
    INTERVENTIONAL
    Phase
    Phase 2
    Registry status
    Recruiting
    Start
    2016-10
    Completion
    2027-04-30
    Enrolment
    150

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

    ConditionRenal Insufficiency, Chronic
    Intervention6R-BH4; 6R-BH4 placebo; Folic acid; Exercise training +3 more
    PhasePhase 2
    Registry statusRecruiting

  • Systemic Sclerosis (SSc) Vasculopathy: Improved Clinical Monitoring and Treatment

    NCT02530996 · start 2016-01-01

    Registered record
    Registry ID
    NCT02530996
    Conditions
    Rheumatologic Disease
    Interventions
    BH4; Vasculopathy assessment; Placebo
    Study type
    INTERVENTIONAL
    Phase
    Phase 1 / Phase 2
    Registry status
    Completed
    Start
    2016-01-01
    Completion
    2019-12-31
    Enrolment
    12

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

    ConditionRheumatologic Disease
    InterventionBH4; Vasculopathy assessment; Placebo
    PhasePhase 1 / Phase 2
    Registry statusCompleted