SAPROPTERIN DIHYDROCHLORIDE
Sapropterin dihydrochloride helps patients with Phenylketonuria (PKU) manage high levels of phenylalanine in the blood. It is used for both adults and children at least one month of age who have a specific form of the condition that responds to tetrahydrobiopterin. This medication is used in combination with a diet that restricts phenylalanine intake.
How SAPROPTERIN DIHYDROCHLORIDE Works
This drug works by providing a synthetic version of BH4, a necessary cofactor for the enzyme that converts phenylalanine into tyrosine. By activating residual enzyme activity, it improves the body's ability to metabolize phenylalanine and reduces its levels in the blood.
Details
- Status
- Prescription
- First Approved
- 2019-05-10
- Routes
- ORAL
- Dosage Forms
- POWDER, TABLET
SAPROPTERIN DIHYDROCHLORIDE Approval History
What SAPROPTERIN DIHYDROCHLORIDE Treats
2 indicationsSAPROPTERIN DIHYDROCHLORIDE is approved for 2 conditions since its original approval in 2019. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Hyperphenylalaninemia
- Phenylketonuria
SAPROPTERIN DIHYDROCHLORIDE Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Direct competitors
Same target(s) AND same indication — head-to-head.
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
Drugs Similar to SAPROPTERIN DIHYDROCHLORIDE
3 of 4FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
4 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT04054973 | H00017202 | Ph 2 | withdrawn | L-arginine Study for Persistent Symptoms of Schizophrenia |
| NCT00789568 | QTC-001 | Ph 1 | completed | A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects |
| NCT00838435 PKU-015 results posted | PKU-015 | Ph 3 | completed | Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU |
| NCT01114737 results posted | PKU-016 PKU Ascend | Ph 3 | completed | Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
SAPROPTERIN DIHYDROCHLORIDE FDA Label Details
Indications & Usage
SAPROPTERIN DIHYDROCHLORIDE is indicated for the treatment of Hyperphenylalaninemia; Phenylketonuria.
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ZELVYSIA
Full clinical data, patents, trials, and competitive landscape for sapropterin dihydrochloride.
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.