Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
sapropterin dihydrochloride + vitamin C
- Development stage
- Unknown
- Lead developer
- BioMarin
- Modality
- Recombinant Proteins and Enzymes, Small Molecules
- Administration
- Oral
01
Overview
Sapropterin dihydrochloride is a synthetic form of tetrahydrobiopterin (BH4), an essential cofactor for the enzyme phenylalanine hydroxylase (PAH). It is used as an adjunct to a phenylalanine-restricted diet to lower blood phenylalanine levels in patients with phenylketonuria (PKU), a genetic disorder characterized by deficient PAH activity. By providing exogenous BH4, sapropterin can activate residual PAH enzyme activity and enhance the conversion of phenylalanine to tyrosine, thereby reducing toxic accumulation of phenylalanine in the blood[1][3][6]. Vitamin C (ascorbic acid) is not part of standard PKU therapy but may be co-administered for general nutritional support; it acts as an antioxidant and supports various enzymatic processes. This combination does not represent a standard fixed-dose pharmaceutical product.
- Brand names
- KuvanJavygtor
- Other names
- tetrahydrobiopterinBH4BH-4BH 4ascorbic acid
02
Targets
TH (Tyrosine hydroxylase)NOS3 (eNOS)PAH (Phenylalanine hydroxylase)TPH1 (Tryptophan 5-hydroxylase 1)
Beyond the preview
Go deeper on sapropterin dihydrochloride + vitamin C.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Full profile accessExplore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Full profile accessTrace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Full profile accessExplore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Full profile accessCompare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Full profile accessConnect source evidence and development news to your research questions.
- Publications
- News
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on sapropterin dihydrochloride + vitamin C.