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SAPROPTERIN
SynDRA canonical compound SYN0023616 — structure-anchored drug synonym record
| Clinical phase | Launched |
|---|
| Approval status | Approved · Rx/OTC |
|---|
| Primary target(s) | TH |
|---|
| Mechanism of action | Phenylalanine 4-hydroxylase stimulant |
|---|
| Indication | hyperphenylalaninemia (HPA) |
|---|
| Disease area | metabolism |
|---|
All known targets
- PAH
- TH
- DBH
- NOS1
- NOS3
- TPH1
Mechanisms of action (all sources)
- Phenylalanine 4-hydroxylase stimulant
- PAH ACTIVATOR
- Phenylalanine-4-hydroxylase activator
- TH COFACTOR
- PAH MODULATOR
Bioactivity evidence (ChEMBL activities table — not curator-asserted)
- ATAD5
- BAZ2B
- CBX1
- EHMT2
- GBA1
- GMNN
- HDAC6
- KDM4A
- PABPC1
- PIN1
- PLK1
- RGS4
- TDP1
- VDR
Screening evidence (raw PubChem HTS — uncurated, known false-positive rate)
- ABC3G
- ADCY9
- B2CL1
- GPX1
- GPX4
- MRGPRX1
- PABP1
Drug classes
- P-Glycoprotein Inhibitors
- Breast Cancer Resistance Protein Inhibitors
- Phenylalanine Hydroxylase Activators
- Phenylalanine Hydroxylase Activator
ATC classes
- A16A · OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
Cross-references
Synonyms
- Javygtor
- KUVAN
- Kuvan
- SAPROPTERIN DIHYDROCHLORIDE
- ZELVYSIA
- Zelvysia
- sapropterin hydrochloride
- tetrahydrobiopterin
- sapropterin dihydrochloride
- kuvan
- sapropterin HCl
- Sapropterine
- Sapropterina
- 6r-bh4
- Dapropterin
- R-thbp
- Tetrahydrobiopterin
- 5,6,7,8-tetrahydrobiopterin
- BRN 0544742
- CHEBI:15372
- 5,6,7,8-erythro-tetrahydrobiopterin
- Phenoptin
- Sapropterin hydrochloride
- Biopten (TN)
- R-THBP
- 6R-BH4
- (6R)-L-erythro-tetrahydrobiopterin
- BH4
- EGX657432I
- Sapropterinum
- 6R-5,6,7,8-Tetrahydrobiopterin
- 6R-L-5,6,7,8-Tetrahydrobiopterin
- sapropterine
- CHEBI:59560
- RefChem:56674
- A16AX07
- Sapropterin [INN]
- Sapropterin [MI]
Reported adverse events
- Headache
- Hypersensitivity
- Cough
- Nasal congestion
- Vomiting
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