Friday, July 10, 2026

Drug profile · INN

sapropterin

sapropterin (KUVAN) is a phenylalanine-4-hydroxylase activator. Associated with BioMarin Pharmaceutical Australia Pty Ltd.

EMA: approved TGA: approved Alimentary tract and metabolism (A16) A16AX07
Patents linked 0
Pipeline programs 22
News articles 0
Drug details — sapropterin
Class
Alimentary tract and metabolism (A16)
Route
Patents
0
Programs
22
Data quality
0.89

Quick answer

sapropterin (KUVAN) is a phenylalanine-4-hydroxylase activator. Associated with BioMarin Pharmaceutical Australia Pty Ltd.

Key facts

INN sapropterin
Brand KUVAN
Mechanism Phenylalanine-4-hydroxylase activator
Therapeutic class Alimentary tract and metabolism (A16)
ATC code A16AX07

Mechanism of action

Phenylalanine-4-hydroxylase activator

Approval history

Authority Status Date
EMA approved
TGA approved

Pipeline programs

  1. PKU-003 Phase 3 · BioMarin Pharmaceutical Australia · Phenylketonurias
  2. PTC923-PKU-301 Phase 3 · PTC THERAPEUTICS · Phenylketonuria
  3. Sapropterin Dihydrochloride Phase 3 · BioMarin Pharmaceutical Australia · Phenylketonurias
  4. Sapropterin dihydrochloride Phase 3 · BioMarin Pharmaceutical Australia · Phenylketonuria
  5. sapropterin Phase 3 · BioMarin Pharmaceutical Australia · Behavioral Manifestations of Lesch-Nyhan Disease
  6. 6R BH4 Phase 2 · BioMarin Pharmaceutical Australia · Kidney Disease
  7. 6R-BH4 Phase 2 · BioMarin Pharmaceutical Australia · Isolated Systolic Hypertension
  8. 6R-BH4 Phase 2 · BioMarin Pharmaceutical Australia · Coronary Artery Disease

Related & competitor drugs

  1. agalsidase alfa Alimentary tract and metabolism (A16)
  2. alogliptin Alimentary tract and metabolism (A10)
  3. alogliptin benzoate Alimentary tract and metabolism (A10)
  4. aprepitant Alimentary tract and metabolism (A04)
  5. budesonide Alimentary tract and metabolism (A07)
  6. canagliflozin Alimentary tract and metabolism (A10)

Frequently asked questions

What is sapropterin?

sapropterin (KUVAN) is a phenylalanine-4-hydroxylase activator. Associated with BioMarin Pharmaceutical Australia Pty Ltd.

What is the brand name for sapropterin?

KUVAN is a marketed brand name for sapropterin. Check FDA Drugs@FDA for the current US label holder and prescribing information.