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sapropterin

Trade name: kuvan

Small moleculeapprovedOrphan Drug FDA

Approved

Dec 13, 2007

Sapropterin dihydrochloride, the active pharmaceutical ingredient in Kuvan Tablets, is a synthetic preparation of the dihydrochloride salt of naturally occurring tetrahydrobiopterin (BH4). Kuvan is indicated to reduce blood phenylalanine (Phe) levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive Phenylketonuria (PKU). Kuvan is to be used in conjunction with a Phe-restricted diet. Kuvan has received orphan drug designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMEA). Kuvan is a synthetic form of BH4, the cofactor for the enzyme phenylalanine hydroxylase (PAH). PAH hydroxylates Phe through an oxidative reaction to form tyrosine. In patients with PKU, PAH activity is absent or deficient. Treatment with BH4 can activate residual PAH enzyme, improve the normal oxidative metabolism of Phe, and decrease Phe levels in some patients. — NCATS

Clinical trial activity

62 trials · 45 clinical orgs · 6 marketing orgs

Phase 1
11
Phase 2
33
Phase 3
12
Phase 4
5

Earliest trial started Dec 1, 2004 (NCT00208780)

Timeline

2000s

  1. Jan 1, 2004

    Earliest Phase 1 Sponsor(trial)

  2. Jan 1, 2004

    Biomarin — Earliest Phase 2 Sponsor(trial)

  3. Jan 29, 2004

    Orphan Drug Designation

  4. Jan 1, 2005

    Biomarin — Earliest Phase 3 Sponsor(trial)

  5. Dec 13, 2007

    Biomarin — Earliest FDA Approval

  6. Dec 13, 2007

    Biomarin — NDA Organization

2010s

  1. Dec 5, 2016

    Par Pharmaceuticals — Marketing Organization

  2. Dec 21, 2017

    Dr. Reddy's Laboratories — Marketing Organization

  3. May 10, 2019

    Endo Pharmaceuticals — Marketing Organization

  4. May 10, 2019

    Bristol-Myers Squibb — Marketing Organization

2020s

  1. Aug 18, 2022

    Annora Pharma — Marketing Organization

Indications

Approved for

Studied for

Mechanism of action

Approval history

  • approvedPriority reviewDec 13, 2007

Chemistry & pharmacology

Loading structure…

SMILES

C[C@H](O)[C@H](O)[C@H]1CNc2nc(N)[nH]c(=O)c2N1
Mol. weight
241.25 g/mol
Lipinski Ro5
1 violation(s)
Rule of 3
No
Chirality
Single Stereoisomer
Inorganic
No
Polymer
No
Delivery
Oral
Availability
Prescription Only

Oral

Yes

Parenteral

No

Topical

No

First in class

Sources

Also known as

  • 1-butanone, 1-(2,4,5-trihydroxyphenyl)
  • 2,4,5-trihydroxybutyrophenone
  • 2',4',5'-trihydroxybutyrophenone
  • 2-amino-6-(1,2-dihydroxypropyl)-5,6,7,8-tetrahydoro-4(1h)-pteridinone
  • 2-amino-6-(1,2-dihydroxypropyl)-5,6,7,8-tetrahydoro-4(1h)-pteridinone
  • 5,6,7,8-erythro-tetrahydrobiopterin
  • 5,6,7,8-tetrahydrobiopterin
  • 5,6,7,8-tetrahydrobiopterin
  • 5,6,7,8-tetrahydrobiopterin
  • 5,6,7,8-tetrahydrobiopterin
  • 5,6,7,8-tetrahydrobiopterin, (s-(r*,s*))-isomer
  • 5,6,7,8-tetrahydrodictyopterin
  • 5,6,7,8-tetrahydro-l-erythrobiopterin
  • (−)-(6r)-2-amino-6-((1r,2s)-1,2-dihydroxypropyl)-5,6,7,8-tetrahydro-4(3h)-pteridinone
  • (−)-(6r)-2-amino-6-((1r,2s)-1,2-dihydroxypropyl)-5,6,7,8-tetrahydro-4(3h)-pteridinone
  • 6r-5,6,7,8-tetrahydrobiopterin
  • 6r-5,6,7,8-tetrahydrobiopterin
  • 6r-bh4
  • 6r-bh4
  • 6r-bh4
  • 6r-bh4
  • 6r-l-5,6,7,8-tetrahydrobiopterin
  • 6r-l-5,6,7,8-tetrahydrobiopterin
  • (6r)-l-erythro-5,6,7,8-tetrahydrobiopterin
  • (6r)-l-erythro-5,6,7,8-tetrahydrobiopterin
  • 6r-l-erythro-5,6,7,8-tetrahydrobiopterin
  • (6r)-l-erythro-tetrahydrobiopterin
  • (6r)-l-erythro-tetrahydrobiopterin
  • bph4
  • daproterin
  • daproterin
  • d-threo-tetrahydrobiopterin
  • fenoptin
  • kuvan
  • kuvan
  • kuvan
  • kuvan
  • kuvan
  • kuvan
  • phenoptin
  • phenoptin
  • phenoptin
  • phenylalanine hydroxylase cofactor
  • ravpal
  • r-thbp
  • r-thbp
  • sapropterin
  • sapropterin
  • sapropterin
  • sapropterin
  • sapropterin
  • sapropterin
  • sapropterin
  • sapropterin
  • sapropterin
  • sapropterina
  • sapropterina
  • sapropterin dihydrochloride
  • sapropterin dihydrochloride
  • sapropterin dihydrochloride
  • sapropterin dihydrochloride
  • sapropterin dihydrochloride
  • sapropterin dihydrochloride
  • sapropterin dihydrochloride
  • sapropterin hcl
  • sapropterin hydrochloride
  • sapropterin hydrochloride
  • sapropterin hydrochloride
  • sapropterin hydrochloride
  • sapropterin hydrochloride
  • sapropterinum
  • sapropterinum
  • saproterin dihydrochloride
  • sun-0588 (shiratori)
  • sun-0588 (shiratori)
  • t-1401 (biomarin)
  • t-1401 (biomarin)
  • tetrahydro-6-biopterin
  • tetrahydrobiopterin
  • tetrahydrobiopterin
  • tetrahydrobiopterin
  • tetrahydrobiopterin
  • tetrahydrobiopterin
  • thbp
  • trihydroxybutyrophenone

Clinical trials

NCT IDPhaseStart dateSponsor(s)
NCT06987773Phase 1Jun 30, 2025Relief Therapeutics
NCT06481709Phase 1Jul 30, 2024Relief Therapeutics
NCT06396637Phase 2Apr 30, 2024Innovent Biologics (Suzhou) Co. Ltd., Sun Yat-Sen University
NCT05299203Phase 1May 31, 2022Sichuan University
NCT04054973Phase 2Aug 14, 2019University of Massachusetts
NCT03493412Phase 4Aug 31, 2018University of Nebraska
NCT02947750Phase 2Oct 1, 2016Emory University, National Heart, Lung, and Blood Institute (NHLBI)
NCT02677870Phase 4May 1, 2016Biomarin, Case Western Reserve University
NCT02774226Phase 2Sep 1, 2015Augusta University
NCT02353312N/AMar 1, 2015Biomarin, Brown University, Department of Veteran Affairs
NCT01977820Phase 2Feb 1, 2014Merck KGaA
NCT02042196Early Phase 1 (Phase 1)Dec 1, 2013National Heart, Lung, and Blood Institute (NHLBI), University of Colorado, University of Colorado, Denver
NCT01965912Phase 4Oct 1, 2013Biomarin
NCT01806051N/AMar 1, 2013Biomarin, University of Southern California
NCT01732471Phase 3Nov 1, 2012Merck KGaA
NCT01706965Phase 1/Phase 2 (Phase 2)Oct 1, 2012Columbia University, Stanley Medical Research Institute
NCT01617070Phase 4May 1, 2012Biomarin, University of Southern California
NCT01560286Phase 2May 1, 2012Biomarin
NCT01439555Phase 2Nov 1, 2011University of Massachusetts
NCT01456286Phase 2/Phase 3 (Phase 3)Oct 1, 2011University of Barcelona
Showing 20 of 62 trials
Page 1 / 4

Organizations

Research & Development (45)

OrganizationOrg typeTrialsAs lead sponsorPhasesEarliest year
BiomarinFor profit352152004
Emory UniversityAcademic/Hospital4422004
Merck KGaAFor profit3332010
National Heart, Lung, and Blood Institute (NHLBI)Government3022011
American Heart AssociationAcademic/Hospital2022004
Augusta UniversityAcademic/Hospital2222010
Merck SeronoFor profit2022010
National Institutes of Health (NIH)Government2022008
Relief TherapeuticsFor profit2212024
The Children's Health CouncilAcademic/Hospital2222009
University of MassachusettsAcademic/Hospital2212011
University of Southern CaliforniaAcademic/Hospital2222012
University of Texas at HoustonAcademic/Hospital2212010
Brown UniversityAcademic/Hospital1012015
Case Western Reserve UniversityAcademic/Hospital1112016
Children's National Medical CenterAcademic/Hospital1112011
Columbia UniversityAcademic/Hospital1112012
Department of Veteran AffairsGovernment1112015
Food and Drug AdministrationGovernment1112005
Georgetown UniversityAcademic/Hospital1012011
45 organizations
Page 1 / 3

Marketing (8)

OrganizationOrg typeRelationshipDate
Annora PharmaFor profitMKTGAug 18, 2022
BiomarinFor profitNDADec 13, 2007
Bristol-Myers SquibbFor profitMKTGMay 10, 2019
Dr. Reddy's LaboratoriesFor profitMKTGDec 21, 2017
Dr. Reddy's LaboratoriesFor profitSYNDec 17, 2018
Dr. Reddy's LaboratoriesFor profitSYNMar 30, 2021
Endo PharmaceuticalsFor profitMKTGMay 10, 2019
Par PharmaceuticalsFor profitMKTGDec 5, 2016