Horizon Pharma plc to Present Data on RAVICTI(R) (glycerol phenylbutyrate) Oral Liquid for Urea Cycle Disorder Patients Aged Two Months to Two Years
DUBLIN, Ireland, March 22, 2017 (GLOBE NEWSWIRE) -- Horizon Pharma plc (NASDAQ:HZNP), a biopharmaceutical company focused on improving patients' lives by identifying, developing, acquiring and commercializing differentiated and accessible medicines that address unmet medical needs, today announced the presentation of new data on the use of RAVICTI® (glycerol phenylbutyrate) Oral Liquid in children living with urea cycle disorders (UCD) two months to two years of age. These data will be presented on Thursday, March 23 at the American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting in Phoenix, Arizona.
The study results are from two clinical trials that enrolled children with UCDs aged two months to less than two years. Study participants were predominantly on relatively stable doses of sodium phenylbutyrate and switched to equivalent doses of RAVICTI. The primary outcomes measured in the study were targeted mean normalized plasma ammonia of less than 100 μmol/L, hyperammonemic crises (HACs) and adverse events (AEs). Safety and efficacy was also evaluated after patients transitioned to RAVICTI.
"The RAVICTI development program is part of our overall approach to reinvest in our medicines for people living with rare diseases," said Jeffrey W. Sherman, M.D., FACP, executive vice president, research and development and chief medical officer, Horizon Pharma plc. "While UCDs are very rare and can be devastating, continued advancements in diagnosis and ongoing studies evaluating management options are helping improve the long-term outcome of people living with the disorder."
RAVICTI is currently indicated for use as a nitrogen-binding agent for chronic management of adult and pediatric patients two years of age or greater with UCDs who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. RAVICTI is not indicated for patients less than two years of age. In June 2016, Horizon submitted a supplemental New Drug Applications (sNDA) with the U.S. Food and Drug Administration (FDA) seeking to expand the approved age range for RAVICTI to include children as young as two months of age.
The poster presentation (abstract #2873; poster #71), entitled "Safety and Efficacy of Glycerol Phenylbutyrate for Management of Urea Cycle Disorders in Patients Aged 2 Months to 2 Years," will be presented on Thursday, March 23, 2017 from 10:00 a.m. - 11:30 a.m. at the ACMG Annual Meeting.