Trial Profile
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)
Status:
Completed
Phase of Trial:
Phase IV
Latest Information Update: 11 Jul 2024
Price :
$35
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At a glance
- Drugs Glycerol phenylbutyrate (Primary)
- Indications Inborn urea cycle disorders
- Focus Therapeutic Use
- Sponsors Horizon Pharma
Most Recent Events
- 30 Jan 2019 The European Commission has approved RAVICTI (glycerol phenylbutyrate) for patients from birth to two months suffering from a urea cycle disorder following a positive CHMP opinion from the European Medicines Agency (EMA), based on the data from this study.
- 27 Dec 2018 According to a Horizon Pharma media release, the U.S. Food and Drug Administration (FDA) has approved a supplemental new drug application (sNDA) to expand the age range for RAVICTI (glycerol phenylbutyrate) Oral Liquid to include infants younger than two months of age living with a urea cycle disorder (UCD).
- 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.