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Drug ReportsSapropterin
Kuvan(sapropterin)
Kuvan, Sapropterin (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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Clinical
Drug
Target
Variants
Financial
Trends
Safety
Events Timeline
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3M
6M
YTD
1Y
2Y
5Y
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FDA approval date
EMA approval date
Patent expiration date
Study first post date
Last update post date
Start date
Primary completion date
Completion date
Results first post date
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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, Sapropterin, Zelvysia
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
javygtorANDA2024-10-22
kuvanNew Drug Application2024-08-26
sapropterin dihydrochlorideANDA2025-11-14
zelvysiaANDA2025-04-30
Indications
FDA
EMA
Indication
Ontology
MeSH
ICD-10
phenylketonurias—D010661E70.0
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
92 clinical trials
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Indications Phases 4
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
PhenylketonuriasD010661—E70.013741631
Indications Phases 3
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
Autistic disorderD001321EFO_0003758F84.0—21——2
Oral manifestationsD009912———11——1
Lesch-nyhan syndromeD007926Orphanet_510E79.1—11——1
Indications Phases 2
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
HypertensionD006973EFO_0000537I1012———3
Peripheral arterial diseaseD058729EFO_0004265—11———2
Intermittent claudicationD007383EFO_0003876I73.911———2
Peripheral vascular diseasesD016491EFO_0003875I73.911———2
Chronic renal insufficiencyD051436—N18—1———1
Kidney diseasesD007674EFO_0003086N08—1———1
AlbuminuriaD000419EFO_0004285R80.9—1———1
Coronary diseaseD003327———1———1
Coronary artery diseaseD003324—I25.1—1———1
Myocardial ischemiaD017202EFO_1001375I20-I25—1———1
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Indications Phases 1
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
Pulmonary arterial hypertensionD000081029——1————1
Familial primary pulmonary hypertensionD065627—I27.01————1
Indications Without Phase
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
Behavior and behavior mechanismsD001520——————11
Developmental disabilitiesD002658EFO_0003852F89————11
Autism spectrum disorderD000067877—F84.0————11
Heart failureD006333EFO_0003144I50————11
Cardiovascular diseasesD002318EFO_0000319I98————11
Epidemiology
Epidemiological information for investigational and approved indications
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Drug
General
Drug common nameSapropterin
INNsapropterin
Description
Sapropterin is a tetrahydropterin that is 2-amino-5,6,7,8-tetrahydropteridin-4(3H)-one in which a hydrogen at position 6 is substituted by a 1,2-dihydroxypropyl group (6R,1'R,2'S-enantiomer). It has a role as a coenzyme, a diagnostic agent, a human metabolite and a cofactor.
Classification
Small molecule
Drug classpteridine derivatives
Image (chem structure or protein)Loading
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 CID135398654
DrugBankDB00360
UNII IDEGX657432I (ChemIDplus, GSRS)
Target
Agency Approved
PAH
PAH
Organism
Homo sapiens
Gene name
PAH
Gene synonyms
NCBI Gene ID
5053
Protein name
phenylalanine-4-hydroxylase
Protein synonyms
Phe-4-monooxygenase, phenylalanine 4-monooxygenase
Uniprot ID
Q8TC14
Mouse ortholog
Pah (18478)
phenylalanine-4-hydroxylase (Q91WV1)
Alternate
No data
Variants
No data
Financial
Revenue by drug
$
€
£
â‚£
Kuvan – BioMarin Pharmaceutical
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Estimated US medical usage
No data
Trends
PubMed Central
Top Terms for Disease or Syndrome:
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Additional graphs summarizing 17,751 documents
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Safety
Black-box Warning
No Black-box warning
Adverse Events
Top Adverse Reactions
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5,572 adverse events reported
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