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Glycerol phenylbutyrate - Horizon Pharma

Drug Profile

Glycerol phenylbutyrate - Horizon Pharma

Alternative Names: GT4P; HPN-100; Ravicti

Latest Information Update: 11 Feb 2019

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At a glance

  • Originator Ucyclyd Pharma
  • Developer Horizon Pharma; National Jewish Health
  • Class Hepatoprotectants; 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
  • Phase I/II Cystic fibrosis
  • No development reported Hepatic encephalopathy

Most Recent Events

  • 27 Dec 2018 Registered for Inborn urea cycle disorders (In infants, In neonates) in USA (PO)
  • 18 Dec 2018 Registered for Inborn urea cycle disorders (Adjunctive treatment, In adolescents, In children, In infants, In adults, In neonates) in European Union (PO)
  • 18 Dec 2018 Registered for Inborn urea cycle disorders (Adjunctive treatment, In adolescents, In children, In infants, In neonates, In adults) in Norway, Liechtenstein, Iceland (PO)
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