Drug Profile


Alternative Names: 6R-BH4; BH4; Biopten; Kuvan; Phenoptin; Sapropterin dihydrochloride; SUN-0588; SUN0588r; Tetrahydrobiopterin

Latest Information Update: 05 Oct 2017

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At a glance

  • Originator Asubio Pharma; Daiichi Sankyo Company
  • Developer Asubio Pharma; BioMarin Pharmaceutical; Daiichi Sankyo Company; John Hopkins University
  • Class Anxiolytics; Nootropics; Pterins; Small molecules
  • Mechanism of Action Nitric oxide synthase stimulants; Phenylalanine hydroxylase stimulants
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Phenylketonuria
  • New Molecular Entity Yes

Highest Development Phases

  • Marketed Phenylketonuria
  • Phase II Isolated systolic hypertension
  • Discontinued Coronary disorders; Hypertension; Kidney disorders; Peripheral arterial disorders; Pulmonary hypertension; Sickle cell anaemia; Vascular disorders

Most Recent Events

  • 19 Sep 2017 Sapropterin receives Orphan Drug status for Phenylketonuria in Australia
  • 17 Aug 2017 BioMarin Pharmaceuticals terminates a phase II trial in Isolated systolic hypertension (In adults, In the elderly) as no subjects were enrolled in the study in USA (PO) (NCT00802893)
  • 13 Apr 2017 BioMarin Pharmaceutical has patent protection for Sapropterin in Europe
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