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news.Health Canada has accepted Hyperion Therapeutics' new drug submission (NDS) and granted priority review for Ravicti (glycerol phenylbutyrate) Oral Liquid indicated to treat urea cycle disorders (UCDs).
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In Canada, standard review takes 18 months or more and priority review, which is granted to promising medicines that address life-threatening or severely debilitating conditions, shortens the review time to about six months.
Ravicti is indicated for use as a nitrogen-binding agent for chronic management of adult and pediatric patients >=2 years of age with UCDs who cannot be managed by dietary protein restriction and/or amino acid supplementation alone
Hyperion president and chief executive officer Donald Santel said the company is happy with the acceptance of the NDS with priority review for RAVICTI and potentially expanding the availability of RAVICTI to UCD patients outside of the US.
"If approved, RAVICTI will be the first treatment approved by Health Canada to treat UCDs," Santel said.
The new drug submission for Ravicti is based on results from ten controlled and uncontrolled clinical trials evaluating the safety and efficacy of the drug in 359 patients across three populations, including 114 UCD patients.
Data from multiple Canadian patients enrolled at the University of Toronto were included in the NDS.
Safety and efficacy of Ravicti is under review by Health Canada and market authorization has not yet been granted.
The drug must be used with dietary protein restriction and, in some cases, dietary supplements (such as essential amino acids, arginine, citrulline, protein-free calorie supplements).