Product Description
Sapropterin dihydrochloride (Kuvan), hereafter referred to as sapropterin, is a synthetic formulation of the active 6R-isomer of tetrahydrobiopterin, a naturally occurring cofactor for phenylalanine hydroxylase. Sapropterin is the first non-dietary treatment for patients with PKU (Sourced from: https://pubmed.ncbi.nlm.nih.gov/19323589/)
Mechanisms of Action: NOS1 Inhibitor
Novel Mechanism: No
Modality: Small Molecule
Route of Administration: Oral
FDA Designation: None *
Approval Status: Approved
Approved Countries: Argentina | Australia | Austria | Belgium | Brazil | Bulgaria | Canada | Chile | Colombia | Croatia | Czech | Denmark | Ecuador | Estonia | European Medicines Agency | Finland | France | Germany | Greece | Hungary | Iceland | Ireland | Israel | Italy | Japan | Korea | Latvia | Lithuania | Luxembourg | Netherlands | New Zealand | Norway | Peru | Poland | Portugal | Romania | Russia | Slovakia | Slovenia | Spain | Sweden | Switzerland | Taiwan | Turkey | Ukraine | United Kingdom | United States | Venezuela
Approved Indications: None
Known Adverse Events: None
Company: Biomarin
Company Location: NOVATO CA 94949
Company CEO: Jean-Jacques Bienaimé
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic: China, United States
Active Clinical Trial Count: 2
Highest Development Phases
Phase 2: Adenocarcinoma|Pancreatic Cancer
Phase 1: Intermittent Claudication|Peripheral Arterial Disease|Peripheral Vascular Diseases
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| ATLAS | P1 |
Recruiting |
Peripheral Arterial Disease|Peripheral Vascular Diseases|Intermittent Claudication |
2025-07-01 |
|
| SYSUCC-PDAC | P2 |
Recruiting |
Pancreatic Cancer|Adenocarcinoma |
2025-04-30 |