KUVAN (sapropterin dihydrochloride)
Kuvan (sapropterin dihydrochloride) is a phenylalanine hydroxylase activator indicated to reduce blood phenylalanine (Phe) levels. It is used in adult and pediatric patients one month of age and older who have hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive Phenylketonuria (PKU). This medication is administered in conjunction with a Phe-restricted diet to manage the condition.
How KUVAN Works
Kuvan is a synthetic form of BH4, which serves as the cofactor for the enzyme phenylalanine hydroxylase (PAH). In a healthy system, PAH converts phenylalanine into tyrosine, but this enzyme activity is deficient or absent in patients with PKU. Treatment with this synthetic cofactor can activate residual PAH enzyme activity, improving the oxidative metabolism of phenylalanine and decreasing blood Phe levels.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2007-12-13
- Patent Cliff
- 2033
- Revenue
- $22M (Q4-2025)
- Routes
- ORAL
- Dosage Forms
- POWDER, TABLET
KUVAN Approval History
What KUVAN Treats
2 indicationsKUVAN is approved for 2 conditions since its original approval in 2007. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Hyperphenylalaninemia
- Phenylketonuria
KUVAN 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 KUVAN
3 of 4FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
9 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT06481709 results posted | 2023-5439 | Ph 1 | completed | Study on Sapropterin Dihydrochloride Oral Suspension in Healthy Subjects |
| NCT04054973 | H00017202 | Ph 2 | withdrawn | L-arginine Study for Persistent Symptoms of Schizophrenia |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 |
| NCT00841100 results posted | 20080675 | Ph 2 | completed | Kuvan Therapy in Phenylketonuria (PKU): The Effect of Blood Phenylalanine Concentration on Kuvan Response |
| NCT01706965 results posted | 6320 | Ph 1, Ph 2 | completed | Kuvan in People With Schizophrenia and Schizoaffective Disorder |
| NCT01617070 results posted | USC Kuvan Melatonin | Ph 4 | completed | Effects of Kuvan on Melatonin Secretion |
| NCT01114737 results posted | PKU-016 PKU Ascend | Ph 3 | completed | Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients |
| NCT01395394 results posted | IRB00046153 | Ph 2 | terminated | Phenylketonuria, Oxidative Stress, and BH4 |
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.
KUVAN FDA Label Details
Indications & Usage
FDA Label (PDF)KUVAN is indicated for the treatment of Hyperphenylalaninemia; Phenylketonuria.
KUVAN Patents & Exclusivity
Patents (6 active)
Pro Intelligence Preview
Deep insights for KUVAN
Revenue Insights
- • Q4-2025: $22M
- • Historical trend analysis
Patent Timeline
- • Cliff: 2033
- • 12 active patents
Trial Analysis
- • 15 total trials
- • Stage: Declining
Competitive Landscape
- • 4 similar drugs
- • Same target/indication analysis
Full approval history • All patents • Revenue trends • Competitor analysis
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment