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: 07 Mar 2019
Price : $35 *
At a glance
- Drugs Glycerol phenylbutyrate (Primary)
- Indications Inborn urea cycle disorders
- Focus Therapeutic Use
- Sponsors Horizon Pharma
- 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).
- 31 Aug 2018 Biomarkers information updated