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Kuvan (SAPROPTERIN)
Kuvan works by activating the enzyme Phenylalanine Hydroxylase to break down phenylalanine.
At a glance
| Generic name | SAPROPTERIN |
|---|---|
| Sponsor | BioMarin |
| Drug class | Phenylalanine Hydroxylase Activator |
| Target | Nitric oxide synthase, brain |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | FDA-approved |
| First approval | 2007 |
| Annual revenue | 300 |
Mechanism of action
Sapropterin dihydrochloride is synthetic form of BH4, the cofactor for the enzyme phenylalanine hydroxylase (PAH). PAH hydroxylates Phe through an oxidative reaction to form tyrosine. In patients with PKU, PAH activity is absent or deficient. Treatment with BH4 can activate residual PAH enzyme activity, improve the normal oxidative metabolism of Phe, and decrease Phe levels in some patients.
Approved indications
- Reduce blood phenylalanine levels in PKU
Common side effects
- Seizures
- Headache
- Diarrhea
- Vomiting
- Cough
- Nasal congestion
- Rhinorrhea
- Pharyngolaryngeal pain
- Gastrointestinal bleeding
- Post-procedural bleeding
- Myocardial infarction
- Respiratory failure
Key clinical trials
- Evaluation of BH4 Responsiveness in Our PKU Patients (PHASE1)
- : Vascular Function in Health and Disease (PHASE1)
- Exercise and NO in HFrEF (EARLY_PHASE1)
- Rhode Island Diastolic Dysfunction - Heart Failure (NA)
- Novel Approaches for Improving Vascular Function in Veterans With HFpEF (PHASE1)
- Study on Sapropterin Dihydrochloride Oral Suspension in Healthy Subjects (PHASE1)
- Study on Sapropterin Dihydrochloride Oral Suspension in Healthy Subjects Under Fed Conditions (PHASE1)
- The Effects of ATLAS Therapy on Nitric Oxide Bioavailability in Patients With Intermittent Claudication (PHASE1)
Patents
| Patent | Expiry | Type |
|---|---|---|
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
| SEC EDGAR | Revenue + earnings |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Kuvan CI brief — competitive landscape report
- Kuvan updates RSS · CI watch RSS
- BioMarin portfolio CI