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Kuvan®
Kuvan® is a Cofactor replacement therapy Small molecule drug developed by Merck KGaA, Darmstadt, Germany. It is currently FDA-approved for Phenylketonuria (PKU) in patients with BH4-responsive mutations. Also known as: Sapropterin dihydrochloride, sapropterin, tetrahydrobiopterin, Diagnostic Tests.
Kuvan is a synthetic form of tetrahydrofolate (BH4) that acts as a cofactor to restore phenylalanine ammonia-lyase (PAL) enzyme activity, enabling the metabolism of phenylalanine in patients with phenylketonuria.
Kuvan is a synthetic form of tetrahydrofolate (BH4) that acts as a cofactor to restore phenylalanine ammonia-lyase (PAL) enzyme activity, enabling the metabolism of phenylalanine in patients with phenylketonuria. Used for Phenylketonuria (PKU) in patients with BH4-responsive mutations.
At a glance
| Generic name | Kuvan® |
|---|---|
| Also known as | Sapropterin dihydrochloride, sapropterin, tetrahydrobiopterin, Diagnostic Tests, sapropterin dihydrochloride |
| Sponsor | Merck KGaA, Darmstadt, Germany |
| Drug class | Cofactor replacement therapy |
| Target | Phenylalanine ammonia-lyase (PAL) enzyme cofactor |
| Modality | Small molecule |
| Therapeutic area | Metabolic/Genetic Disorders |
| Phase | FDA-approved |
Mechanism of action
Kuvan (sapropterin dihydrochloride) is a pteridine compound that serves as a cofactor for the enzyme phenylalanine ammonia-lyase (PAL), which catalyzes the conversion of phenylalanine to tyrosine. In phenylketonuria (PKU), mutations in the PAH gene reduce PAL enzyme activity, leading to toxic accumulation of phenylalanine. By supplementing with synthetic BH4, Kuvan restores sufficient enzyme cofactor levels in responsive patients, allowing phenylalanine metabolism and reducing blood phenylalanine concentrations.
Approved indications
- Phenylketonuria (PKU) in patients with BH4-responsive mutations
Common side effects
- Headache
- Rhinitis
- Diarrhea
- Fever
Key clinical trials
- 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)
- Study of Kuvan Treatment in Adults With GTPCH Deficiency (PHASE1, PHASE2)
- Evaluation of Behavior, Executive Function, Neurotransmitter Function and Genomic Expression Kuvan Nonresponders
- Kuvan®'s Effect on the Cognition of Children With Phenylketonuria (PHASE4)
- Clinical Antenatal Randomised Study to CharactErise Key Roles of TetrahydroFOLate in HyperTensive Pregnancies (NA)
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 |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
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
- Merck KGaA, Darmstadt, Germany portfolio CI
Frequently asked questions about Kuvan®
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Related
- Drug class: All Cofactor replacement therapy drugs
- Target: All drugs targeting Phenylalanine ammonia-lyase (PAL) enzyme cofactor
- Manufacturer: Merck KGaA, Darmstadt, Germany — full pipeline
- Therapeutic area: All drugs in Metabolic/Genetic Disorders
- Indication: Drugs for Phenylketonuria (PKU) in patients with BH4-responsive mutations
- Also known as: Sapropterin dihydrochloride, sapropterin, tetrahydrobiopterin, Diagnostic Tests, sapropterin dihydrochloride