sapropterin Indications/Contra | FAERs | Orange Book | BioActivity |

Stem definitionDrug idCAS RN
2612 62989-33-7

Description:

MoleculeDescription

Molfile Inchi Smiles

Synonyms:

  • sapropterin hydrochloride
  • tetrahydrobiopterin
  • sapropterin
  • sapropterin dihydrochloride
  • kuvan
  • sapropterin HCl
  • Molecular weight: 241.25
  • Formula: C9H15N5O3
  • CLOGP: -1.17
  • LIPINSKI: 1
  • HAC: 8
  • HDO: 6
  • TPSA: 132
  • ALOGS: -2.07
  • ROTB: 2

Drug dosage:

None

Approvals:

DateAgencyCompanyOrphan
Dec. 2, 2008 EMA
Dec. 13, 2007 FDA BIOMARIN PHARM

FDA Adverse Event Reporting System

MedDRA adverse event termLikelihood ratioLikelihood ratio thresholdPatients taking drug having adverse eventPatients taking drug not having adverse eventPatients not taking drug having adverse eventPatients not taking drug not having adverse event
Maternal exposure during pregnancy 95.25 35.42 25 344 17068 4621555
Amino acid level increased 59.78 35.42 8 361 128 4638495
Headache 56.04 35.42 28 341 122345 4516278
Psychomotor hyperactivity 53.52 35.42 12 357 4177 4634446
Dizziness 43.07 35.42 22 347 99849 4538774
Amino acid level decreased 42.07 35.42 5 364 28 4638595
Exposure during pregnancy 39.69 35.42 15 354 32956 4605667
Vomiting 39.32 35.42 22 347 119677 4518946
Aggression 39.25 35.42 12 357 13887 4624736

Pharmacologic Action:

SourceCodeDescription
ATC A16AX07 ALIMENTARY TRACT AND METABOLISM
OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
Various alimentary tract and metabolism products
FDA EPC N0000190483 Phenylalanine Hydroxylase Activator
FDA MoA N0000190482 Phenylalanine Hydroxylase Activators
FDA MoA N0000190113 Breast Cancer Resistance Protein Inhibitors
FDA MoA N0000185503 P-Glycoprotein Inhibitors
CHEBI has role CHEBI:33295 diagnostic agent

Drug Use (View source of the data)

DiseaseRelationSNOMED_IDDOID
Classical phenylketonuria indication 7573000
Neutropenic disorder contraindication 303011007 DOID:1227

Acid dissociation constants calculated using MoKa v3.0.0

Dissociation levelDissociation constantType (acidic/basic)
pKa1 11.23 acidic
pKa2 5.42 Basic

Orange Book patent data (new drug applications)

Formulation strengthTrade nameApplicantApplication numberApproval dateTypeDose formRoutePatent numberPatent expiration datePatent use
100MG KUVAN BIOMARIN PHARM N022181 Dec. 13, 2007 RX TABLET ORAL 7566714 Nov. 17, 2024 FOR REDUCING BLOOD PHENYLALANINE LEVELS IN A HUMAN SUFFERING FROM HYPERPHENYLALANINEMIA
100MG KUVAN BIOMARIN PHARM N022181 Dec. 13, 2007 RX TABLET ORAL 7612073 Nov. 17, 2024 TO REDUCE BLOOD PHENYLALANINE LEVELS IN PATIENTS WITH HYPERPHENYLALANINEMIA DUE TO TETRA HYDROBIOPTERIN RESPONSIVE PHENYLKETONURIA. KUVAN SHOULD BE TAKEN ORALLY WITH FOOD TO INCREASE ABSORPTION
100MG KUVAN BIOMARIN PHARM N022181 Dec. 13, 2007 RX TABLET ORAL 7947681 Nov. 17, 2024 TO REDUCE BLOOD PHENYLALANINE (PHE) LEVELS IN PATIENTS WITH HYPERPHENYLALANINEMIA (HPA)
100MG KUVAN BIOMARIN PHARM N022181 Dec. 13, 2007 RX TABLET ORAL 8067416 Nov. 17, 2024 FOR REDUCING BLOOD PHENYLALANINE LEVELS IN A HUMAN SUFFERING FROM HYPERPHENYLALANINEMIA
100MG KUVAN BIOMARIN PHARM N022181 Dec. 13, 2007 RX TABLET ORAL 9433624 Nov. 17, 2024 METHOD OF USE FOR REDUCING BLOOD PHENYLALANINE LEVELS IN A HUMAN SUFFERING FROM HYPERPHENYLALANINEMIA
100MG KUVAN BIOMARIN PHARM N022181 Dec. 13, 2007 RX TABLET ORAL RE43797 Nov. 17, 2024 TO REDUCE BLOOD PHENYLALANINE (PHE) LEVELS IN PATIENTS WITH HYPERPHENYLALANINEMIA (HPA)
100MG/PACKET KUVAN BIOMARIN PHARM N205065 Dec. 19, 2013 RX POWDER ORAL 7566714 Nov. 17, 2024 METHOD OF USE FOR REDUCING BLOOD PHENYLALANINE LEVELS IN A HUMAN SUFFERING FROM HYPERPHENYLALANINEMIA
100MG/PACKET KUVAN BIOMARIN PHARM N205065 Dec. 19, 2013 RX POWDER ORAL 7612073 Nov. 17, 2024 TO REDUCE BLOOD PHENYLALANINE LEVELS IN PATIENTS WITH HYPERPHENYLALANINEMIA DUE TO TETRA HYDROBIOPTERIN RESPONSIVE PHENYLKETONURIA. KUVAN SHOULD BE TAKEN ORALLY WITH FOOD TO INCREASE ABSORPTION
100MG/PACKET KUVAN BIOMARIN PHARM N205065 Dec. 19, 2013 RX POWDER ORAL 8067416 Nov. 17, 2024 METHOD OF USE FOR REDUCING BLOOD PHENYLALANINE LEVELS IN A HUMAN SUFFERING FROM HYPERPHENYLALANINEMIA
100MG/PACKET KUVAN BIOMARIN PHARM N205065 Dec. 19, 2013 RX POWDER ORAL 9433624 Nov. 17, 2024 METHOD OF USE FOR REDUCING BLOOD PHENYLALANINE LEVELS IN A HUMAN SUFFERING FROM HYPERPHENYLALANINEMIA
100MG/PACKET KUVAN BIOMARIN PHARM N205065 Dec. 19, 2013 RX POWDER ORAL RE43797 Nov. 17, 2024 KUVAN IS INDICATED TO REDUCE BLOOD PHENYLALANINE LEVELS IN PATIENTS WITH HYPERPHENYLALANINEMIA
500MG/PACKET KUVAN BIOMARIN PHARM N205065 Oct. 27, 2015 RX POWDER ORAL 7566714 Nov. 17, 2024 METHOD OF USE FOR REDUCING BLOOD PHENYLALANINE LEVELS IN A HUMAN SUFFERING FROM HYPERPHENYLALANINEMIA
500MG/PACKET KUVAN BIOMARIN PHARM N205065 Oct. 27, 2015 RX POWDER ORAL 7612073 Nov. 17, 2024 TO REDUCE BLOOD PHENYLALANINE LEVELS IN PATIENTS WITH HYPERPHENYLALANINEMIA DUE TO TETRA HYDROBIOPTERIN RESPONSIVE PHENYLKETONURIA. KUVAN SHOULD BE TAKEN ORALLY WITH FOOD TO INCREASE ABSORPTION
500MG/PACKET KUVAN BIOMARIN PHARM N205065 Oct. 27, 2015 RX POWDER ORAL 8067416 Nov. 17, 2024 METHOD OF USE FOR REDUCING BLOOD PHENYLALANINE LEVELS IN A HUMAN SUFFERING FROM HYPERPHENYLALANINEMIA
500MG/PACKET KUVAN BIOMARIN PHARM N205065 Oct. 27, 2015 RX POWDER ORAL 9433624 Nov. 17, 2024 METHOD OF USE FOR REDUCING BLOOD PHENYLALANINE LEVELS IN A HUMAN SUFFERING FROM HYPERPHENYLALANINEMIA
500MG/PACKET KUVAN BIOMARIN PHARM N205065 Oct. 27, 2015 RX POWDER ORAL RE43797 Nov. 17, 2024 KUVAN IS INDICATED TO REDUCE BLOOD PHENYLALANINE LEVELS IN PATIENTS WITH HYPERPHENYLALANINEMIA

Orange Book exclusivity data (new drug applications)

None

Bioactivity Summary:

TargetClassPharosUniProtActionTypeActivity value
(-log[M])
Mechanism
action
Bioact sourceMoA source
Phenylalanine-4-hydroxylase Enzyme ACTIVATOR Ki 5.40 IUPHAR CHEMBL
Nitric oxide synthase, brain Enzyme Kd 5.95 CHEMBL

External reference:

IDSource
4027166 VUID
N0000176128 NUI
C0048897 UMLSCUI
D01798 KEGG_DRUG
432857000 SNOMEDCT_US
d07068 MMSL
753340 RXNORM
4027166 VANDF
432859002 SNOMEDCT_US
012473 NDDF
135398654 PUBCHEM_CID
CHEBI:59560 CHEBI
DB00360 DRUGBANK_ID
EGX657432I UNII
6553 INN_ID
69056-38-8 SECONDARY_CAS_RN
CHEMBL1201774 ChEMBL_ID
CHEMBL1201775 ChEMBL_ID
5276 IUPHAR_LIGAND_ID
C003402 MESH_SUPPLEMENTAL_RECORD_UI

Pharmaceutical products:

ProductCategoryIngredientsNDCFormQuantityRouteMarketingLabel
Kuvan HUMAN PRESCRIPTION DRUG LABEL 1 68135-300 TABLET 100 mg ORAL NDA 19 sections
Kuvan HUMAN PRESCRIPTION DRUG LABEL 1 68135-301 POWDER, FOR SOLUTION 100 mg ORAL NDA 19 sections
Kuvan HUMAN PRESCRIPTION DRUG LABEL 1 68135-482 POWDER, FOR SOLUTION 500 mg ORAL NDA 19 sections