Wednesday, July 8, 2026

Drug profile · INN

sapropterin dihydrochloride

sapropterin dihydrochloride (SAPROPTERIN DIHYDROCHLORIDE) is a phenylalanine-4-hydroxylase activator. Associated with BioMarin Pharmaceutical Australia Pty Ltd. Route of administration: oral. Current US regulatory status: approved.

FDA: approved
US status approved
Patents linked 3
Pipeline programs 20
News articles 0
Drug details — sapropterin dihydrochloride
US status
approved
Class
Route
ORAL
Patents
3
Programs
20
Data quality
0.89

Quick answer

sapropterin dihydrochloride (SAPROPTERIN DIHYDROCHLORIDE) is a phenylalanine-4-hydroxylase activator. Associated with BioMarin Pharmaceutical Australia Pty Ltd. Route of administration: oral. Current US regulatory status: approved.

Key facts

INN sapropterin dihydrochloride
Brand SAPROPTERIN DIHYDROCHLORIDE
Mechanism Phenylalanine-4-hydroxylase activator
Target Phenylalanine-4-hydroxylase
Route ORAL
Formulation POWDER

Mechanism of action

Phenylalanine-4-hydroxylase activator

Primary target: Phenylalanine-4-hydroxylase

Approval history

Authority Status Date
FDA approved

Pipeline programs

  1. Kuvan® Phase 3 · BioMarin Pharmaceutical Australia · Autistic Disorder
  2. PKU-003 Phase 3 · BioMarin Pharmaceutical Australia · Phenylketonurias
  3. PTC923-PKU-301 Phase 3 · PTC THERAPEUTICS · Phenylketonuria
  4. Sapropterin Dihydrochloride Phase 3 · BioMarin Pharmaceutical Australia · Phenylketonurias
  5. Sapropterin dihydrochloride Phase 3 · BioMarin Pharmaceutical Australia · Phenylketonuria
  6. sapropterin Phase 3 · BioMarin Pharmaceutical Australia · Behavioral Manifestations of Lesch-Nyhan Disease
  7. 6R-BH4 (sapropterin dihydrochloride) Phase 2 · BioMarin Pharmaceutical Australia · Hypertension
  8. Sapropterin Phase 2 · BioMarin Pharmaceutical Australia · GTP Cyclohydrolase Deficiency
  1. US 9216178

    Dry blend formulation of tetrahydrobiopterin

    Expires Mon Dec 17 2035 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted
  2. US 8003126

    Stable tablet formulation

    Expires Mon Aug 18 2031 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted
  3. US 7566462

    Stable tablet formulation

    Expires Mon Jul 23 2029 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted

Frequently asked questions

What is sapropterin dihydrochloride?

sapropterin dihydrochloride (SAPROPTERIN DIHYDROCHLORIDE) is a phenylalanine-4-hydroxylase activator. Associated with BioMarin Pharmaceutical Australia Pty Ltd. Route of administration: oral. Current US regulatory status: approved.

What is the brand name for sapropterin dihydrochloride?

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

What is the US approval status of sapropterin dihydrochloride?

sapropterin dihydrochloride has approved status in the United States according to our Drugs@FDA-derived records. Verify on the official FDA label before clinical or commercial decisions.