Horizon Pharma plc made a presentation of new data on Ravicti (glycerol phenylbutyrate) at a recent American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics meeting.
The data resulted from two clinical trials, according to a Horizon Pharma press release. The
participants, children age 2 months to 2 years, were being treated with
sodium phenylbutyrate for urea cycle disorders (UCD). The study switched the participants
and evaluated the results, including safety and efficacy.
development program is part of our overall
approach to reinvest in our medicines for people living with rare
Dr. Jeffrey W. Sherman, Horizon Pharma executive vice president, research and
development and chief medical officer, said in the release. "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."
Horizon is seeking Food and Drug Administration
approval to expand the age range to children ages 2 months and older. It
submitted a supplemental New Drug Applications (sNDA) to the FDA in June 2016.
UCD is a genetic disorder. It is a enzyme deficiency in the
urea cycle, which causes elevated ammonia levels in the blood. The excess
ammonia can cause brain damage, coma or death. It affects approximately 1 in
35,000 in the U.S.
Horizon Pharma presents new data on Ravicti
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