A Switch-Over, Open-Label Study of the Safety, Pharmacokinetics, and Efficacy of HPN-100, Followed by Long-Term Treatment With HPN-100, in Pediatric Subjects Under 6 Years of Age With Urea Cycle Disorders (UCDs)
Phase of Trial: Phase III
Latest Information Update: 10 May 2017
At a glance
- Drugs Glycerol phenylbutyrate (Primary)
- Indications Inborn urea cycle disorders
- Focus Adverse reactions
- Sponsors Hyperion Therapeutics
- 10 May 2017 According to a Horizon Pharma media release, the US FDA has approved the company's supplemental New Drug Application (sNDA) to expand the age range for RAVICTI (glycerol phenylbutyrate) Oral Liquid to people two months of age and older who have urea cycle disorders (UCDs) that cannot be managed by dietary protein restriction and/or amino acid supplementation alone. The approval was based on this and two other studies.
- 29 Jun 2016 According to a Horizon Pharma media release, based on this and other study (700249537) the company has submitted supplemental New Drug Application (sNDA) to the U.S. FDA for glycerol phenylbutyrate, Oral Liquid, to expand the age range of patients from two years of age to two months of age and older for the chronic management of urea cycle disorders (UCDs).
- 30 Nov 2015 According to Horizon Pharma media release, the European Commission has approved glycerol phenylbutyrate oral liquid (RAVICTI) for treatment of urea cycle disorders in patients two months of age and older.
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Table of Contents
- At a glance
- Trial Overview
- Trial Details
- Trial Centres
- Trial History