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Kuvan(sapropterin)
Kuvan (sapropterin) is a small molecule pharmaceutical. Sapropterin was first approved as Kuvan on 2007-12-13. It is used to treat phenylketonurias in the USA. It has been approved in Europe to treat phenylketonurias. The pharmaceutical is active against phenylalanine-4-hydroxylase.
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Commercial
Therapeutic Areas
Therapeutic Area
MeSH
nervous system diseasesD009422
hereditary congenital and neonatal diseases and abnormalitiesD009358
nutritional and metabolic diseasesD009750
Trade Name
FDA
EMA
Kuvan (generic drugs available since 2019-05-10)
Drug Products
FDA
EMA
New Drug Application (NDA)
New Drug Application (NDA)
Abbreviated New Drug Application (ANDA)
Abbreviated New Drug Application (ANDA)
Sapropterin dihydrochloride
Tradename
Company
Number
Date
Products
KUVANBioMarin PharmaceuticalN-022181 RX2007-12-13
1 products, RLD, RS
KUVANBioMarin PharmaceuticalN-205065 RX2013-12-19
2 products, RLD, RS
Labels
FDA
EMA
Brand Name
Status
Last Update
kuvanNew Drug Application2021-02-08
sapropterin dihydrochlorideANDA2022-11-03
Indications
FDA
EMA
Indication
Ontology
MeSH
ICD-10
phenylketonuriasD010661E70.0
Agency Specific
FDA
EMA
No data
Patent Expiration
Patent
Expires
Flag
FDA Information
Sapropterin Dihydrochloride, Kuvan, Biomarin Pharm
92161782032-11-01DP
75664622025-11-16DP
80031262025-11-16
RE437972024-11-17U-1156, U-1590
75667142024-11-17U-989, U-1589
76120732024-11-17U-1010
77279872024-11-17DP
80674162024-11-17U-989, U-1589
83187452024-11-17DP
94336242024-11-17U-1589
ATC Codes
A: Alimentary tract and metabolism drugs
A16: Other alimentary tract and metabolism products in atc
A16A: Other alimentary tract and metabolism products in atc
A16AX: Various alimentary tract and metabolism products
A16AX07: Sapropterin
HCPCS
No data
Clinical
Clinical Trials
70 clinical trials
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Mock data
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Indications Phases 4
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
PhenylketonuriasD010661E70.037941133
Peripheral arterial diseaseD058729EFO_0004265112
Indications Phases 3
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
Autistic disorderD001321EFO_0003758F84.0212
Liver cirrhosisD008103EFO_0001422K74.0111
Portal hypertensionD006975EFO_0000666K76.6111
Oral manifestationsD009912111
Indications Phases 2
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
Chronic obstructive pulmonary diseaseD029424EFO_0000341J44.92114
HypertensionD006973EFO_0000537I10213
Intermittent claudicationD007383EFO_0003876I73.9112
SchizophreniaD012559EFO_0000692F20122
Hematologic diseasesD006402EFO_0005803D75.9111
Kidney diseasesD007674EFO_0003086N0811
AlbuminuriaD000419EFO_0004285R80.911
Coronary artery diseaseD003324I25.111
Sickle cell anemiaD000755EFO_0000697D5711
Chronic renal insufficiencyD051436N1811
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Indications Phases 1
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
Heart failureD006333EFO_0003144I50213
Pulmonary arterial hypertensionD00008102922
Cardiovascular diseasesD002318EFO_0000319I98112
AgingD000375GO_0007568R41.8111
MenopauseD008593EFO_0003922N9511
Peripheral vascular diseasesD016491EFO_0003875I73.911
Heart disease risk factorsD00008274211
VasoconstrictionD01466111
Oxidative stressD018384EFO_100190511
RadiodermatitisD011855L5811
Show 1 more
Indications Without Phase
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
Behavior and behavior mechanismsD00152011
Autism spectrum disorderD000067877F84.011
GastroparesisD018589EFO_1000948K31.8411
Epidemiology
Epidemiological information for investigational and approved indications
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Drug
General
Drug common nameSAPROPTERIN
INNsapropterin
Description
Tetrahydrobiopterin (BH4, THB), also known as sapropterin (INN), is a cofactor of the three aromatic amino acid hydroxylase enzymes, used in the degradation of amino acid phenylalanine and in the biosynthesis of the neurotransmitters serotonin (5-hydroxytryptamine, 5-HT), melatonin, dopamine, norepinephrine (noradrenaline), epinephrine (adrenaline), and is a cofactor for the production of nitric oxide (NO) by the nitric oxide synthases. Chemically, its structure is that of a (dihydropteridine reductase) reduced pteridine derivative (quinonoid dihydrobiopterin).
Classification
Small molecule
Drug classpteridine derivatives
Image (chem structure or protein)
Structure (InChI/SMILES or Protein Sequence)
C[C@H](O)[C@H](O)[C@H]1CNc2nc(N)[nH]c(=O)c2N1
Identifiers
PDB
CAS-ID17528-72-2
RxCUI
ChEMBL IDCHEMBL1201774
ChEBI ID59560
PubChem CID44257
DrugBankDB00360
UNII IDEGX657432I (ChemIDplus, GSRS)
Target
Agency Approved
PAH
PAH
Organism
Homo sapiens
Gene name
PAH
Gene synonyms
NCBI Gene ID
Protein name
phenylalanine-4-hydroxylase
Protein synonyms
Phe-4-monooxygenase, phenylalanine 4-monooxygenase
Uniprot ID
Mouse ortholog
Pah (18478)
phenylalanine-4-hydroxylase (Q91WV1)
Alternate
No data
Variants
Clinical Variant
No data
Financial
Kuvan - BioMarin Pharmaceutical
$
£
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Tabular view
Trends
PubMed Central
Top Terms for Disease or Syndrome:
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Additional graphs summarizing 1,134 documents
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Safety
Black-box Warning
No Black-box warning
Adverse Events
Top Adverse Reactions
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267 adverse events reported
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