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saproterin dihydrochloride
Saproterin dihydrochloride is a synthetic form of tetrahydrofolate that acts as a cofactor to enhance phenylalanine ammonia-lyase (PAL) enzyme activity, reducing blood phenylalanine levels in phenylketonuria.
Saproterin dihydrochloride is a synthetic form of tetrahydrofolate that acts as a cofactor to enhance phenylalanine ammonia-lyase (PAL) enzyme activity, reducing blood phenylalanine levels in phenylketonuria. Used for Phenylketonuria (PKU) in patients with residual phenylalanine ammonia-lyase activity.
At a glance
| Generic name | saproterin dihydrochloride |
|---|---|
| Also known as | Kuvan treatment |
| Sponsor | University Hospitals Cleveland Medical Center |
| Drug class | Cofactor replacement therapy |
| Target | Phenylalanine ammonia-lyase (PAL) cofactor |
| Modality | Small molecule |
| Therapeutic area | Metabolic/Genetic Disorder |
| Phase | FDA-approved |
Mechanism of action
Saproterin (also known as BH4 or tetrahydrobiopterin) is an essential cofactor for the enzyme phenylalanine ammonia-lyase, which catalyzes the conversion of phenylalanine to tyrosine. In phenylketonuria (PKU), patients have deficient or dysfunctional PAL enzyme; saproterin supplementation can restore or enhance residual enzyme activity, thereby lowering toxic phenylalanine accumulation. This mechanism is distinct from dietary phenylalanine restriction and allows some PKU patients to achieve better metabolic control and dietary flexibility.
Approved indications
- Phenylketonuria (PKU) in patients with residual phenylalanine ammonia-lyase activity
Common side effects
- Headache
- Rhinitis
- Diarrhea
- Pharyngitis
Key clinical trials
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:
- saproterin dihydrochloride CI brief — competitive landscape report
- saproterin dihydrochloride updates RSS · CI watch RSS
- University Hospitals Cleveland Medical Center portfolio CI