Sapropterin
- Generic Name
- Sapropterin
- Brand Names
- Javygtor, Kuvan, Sapropterin Dipharma
- Drug Type
- Small Molecule
- Chemical Formula
- C9H15N5O3
- CAS Number
- 62989-33-7
- Unique Ingredient Identifier
- EGX657432I
- Background
Sapropterin (tetrahydrobiopterin or BH4) is a cofactor in the synthesis of nitric oxide. It is also essential in the conversion of phenylalanine to tyrosine by the enzyme phenylalanine-4-hydroxylase; the conversion of tyrosine to L-dopa by the enzyme tyrosine hydroxylase; and conversion of tryptophan to 5-hydroxytryptophan via tryptophan hydroxylase.
- Indication
For the treatment of tetrahydrobiopterin (BH4) deficiency.
- Associated Conditions
- Hyperphenylalaninemia
A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels
Phase 3
Completed
- Conditions
- Phenylketonurias
- First Posted Date
- 2005-09-26
- Last Posted Date
- 2009-08-20
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 100
- Registration Number
- NCT00225615
Effects of Tetrahydrobiopterin on Blood Pressure
- First Posted Date
- 2005-09-21
- Last Posted Date
- 2013-11-18
- Lead Sponsor
- Emory University
- Target Recruit Count
- 24
- Registration Number
- NCT00208780
- Locations
- 🇺🇸
Emory University, Atlanta, Georgia, United States
Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels
Phase 3
Completed
- Conditions
- Phenylketonurias
- First Posted Date
- 2005-02-25
- Last Posted Date
- 2014-07-23
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 89
- Registration Number
- NCT00104247
Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria
Phase 2
Completed
- Conditions
- Phenylketonurias
- First Posted Date
- 2005-02-25
- Last Posted Date
- 2007-04-09
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 700
- Registration Number
- NCT00104260
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