An Open Label Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate (GPB; RAVICTI) in Pediatric Subjects Under Two Years of Age With Urea Cycle Disorders (UCDs)
Phase of Trial: Phase IV
Latest Information Update: 27 Feb 2018
At a glance
- Drugs Glycerol phenylbutyrate (Primary)
- Indications Inborn urea cycle disorders
- Focus Therapeutic Use
- Sponsors Horizon Pharma
- 27 Feb 2018 According to a Horizon Pharma media release, based on the data from this trial, the company has submitted a supplemental New Drug Application (sNDA) with the U.S. Food FDA to expand the approved indication for RAVICTI (glycerol phenylbutyrate) Oral Liquid to include infants younger than two months of age living with urea cycle disorders (UCDs). The FDA is expected to complete their review during the second half of 2018.
- 15 Aug 2017 Status changed from active, no longer recruiting to completed.
- 01 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.