Trial Profile
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)
Status:
Completed
Phase of Trial:
Phase III
Latest Information Update: 19 Jul 2024
Price :
$35
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At a glance
- Drugs Glycerol phenylbutyrate (Primary)
- Indications Inborn urea cycle disorders
- Focus Adverse reactions
- Sponsors Hyperion Therapeutics
Most Recent Events
- 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).
- 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).