Glycerol phenylbutyrate

Drug Profile

Glycerol phenylbutyrate

Alternative Names: Glyceryl tri-(4-phenylbutyrate); GT4P; HPN-100; Ravicti

Latest Information Update: 17 May 2017

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At a glance

  • Originator Ucyclyd Pharma
  • Developer Horizon Pharma
  • Class Phenylbutyrates; Small molecules
  • Mechanism of Action Ammonia scavengers
  • Orphan Drug Status

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

    Yes - Inborn urea cycle disorders; Hepatic encephalopathy; Spinal muscular atrophy
  • New Molecular Entity No

Highest Development Phases

  • Marketed Inborn urea cycle disorders
  • No development reported Hepatic encephalopathy

Most Recent Events

  • 28 Apr 2017 Registered for Inborn urea cycle disorders (Adjunctive treatment, In infants) in USA (PO)
  • 22 Dec 2016 Horizon Pharma receives patent allowance for glycerol phenylbutyrate in USA
  • 15 Dec 2016 Biomarkers information updated
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