Glycerol phenylbutyrate

Drug Profile

Glycerol phenylbutyrate

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

Latest Information Update: 28 Dec 2016

Price : $50

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

  • 22 Dec 2016 Horizon Pharma receives patent allowance for glycerol phenylbutyrate in USA
  • 07 Dec 2016 Swedish Orphan Biovitrum obtains rights to distribute and market glycerol phenylbutyrate in Europe, including United Kingdom, Germany, France, Italy and Spain
  • 03 Nov 2016 Launched for Inborn urea cycle disorders (Adjunctive treatment, In adolescents, In children, In adults) in Canada (PO)
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