sapropterin (kuvan) Report issue

Small molecule Orphan Drug FDA Approved FDA First in Class Priority Review FDA
AACT ChEMBL DrugBank Drug Central Drugs@FDA KEGG MeSH PubChem repoDB

Active Ingredient History

NOW
  • Now
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

Chemistry

More Chemistry

Pharmacology

  • Mechanism of Action:
  • Multi-specific: Missing data
  • Black Box: No
  • Availability: Prescription Only
  • Delivery Methods: Oral
  • Pro Drug: No

Drug Pricing (per unit)

United States

$18.3378 - $187.3580

Australia

$21.3471 - $106.9692
More Pricing Detail

Note: This drug pricing data is preliminary, incomplete, and may contain errors.

Alternative names

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 | 5,6,7,8-erythro-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-5,6,7,8-tetrahydrobiopterin | 6r-bh4 | 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-tetrahydrobiopterin | bph4 | daproterin | d-threo-tetrahydrobiopterin | fenoptin | kuvan | phenoptin | phenylalanine hydroxylase cofactor | ravpal | r-thbp | sapropterin | sapropterina | sapropterin dihydrochloride | sapropterin hcl | sapropterin hydrochloride | sapropterinum | sun-0588 (shiratori) | t-1401 (biomarin) | tetrahydro-6-biopterin | tetrahydrobiopterin | thbp | trihydroxybutyrophenone

Organizations (45)

Research & Development (41)

Organization Org Type FDA approvals Clinical Trials involvement Org ID Force Sort

Marketing (5)

Organization Org Type FDA approvals Clinical Trials involvement Org ID Force Sort

Indications (25)

Approved Indications (1)


 

Phenylketonurias

Experimental Indications - Clinical Trial Phases 1-4 (25)


 

Aging (Early Phase 1)

Albuminuria (Phase 2)

Alzheimer Disease (Phase 2)

Anemia, Sickle Cell (Phase 2)

Attention Deficit and Disruptive Behavior Disorders (Phase 2/Phase 3)

Autistic Disorder (Phase 2/Phase 3)

Coronary Artery Disease (Phase 2)

Deficiency Diseases (Phase 2)

Dopamine beta-Hydroxylase (Phase 2/Phase 3)

Endothelial Cells (Phase 2)

Hypertension (Phase 2)

Hypertension, Portal (Phase 2/Phase 3)

Intermittent Claudication (Phase 2)

Kidney Diseases (Phase 2)

Kidney Failure, Chronic (Phase 2)

Liver Cirrhosis (Phase 2/Phase 3)

Menopause (Early Phase 1)

Peripheral Arterial Disease (Phase 4)

Phenylketonurias ()

Psychotic Disorders (Phase 1/Phase 2)

Pulmonary Arterial Hypertension (Phase 1)

Pulmonary Disease, Chronic Obstructive (Phase 2)

Renal Insufficiency, Chronic (Phase 2)

Schizophrenia (Phase 2)

Squamous Cell Carcinoma of Head and Neck (Phase 1)

Overview

  • Highest Phase: Phase 4
  • # of Trials: 58
  • # of Trial Organizations: 41

Trials by Phase

Trial Phase Start Date Organizations Indications

Feedback

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