Horizon Pharma Announces FDA Approval to Expand the Age Range for RAVICTI

Horizon Pharma (NASDAQ:HZNP) today announced 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).

As quoted in the press release:

RAVICTI is now FDA-approved for use as a nitrogen-binding agent for chronic management of UCDs in adults and children of all ages who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements. RAVICTI is not indicated for treatment of acute hyperammonemia in patients with UCDs, and its safety and efficacy for the treatment of n-acetylglutamate synthase (NAGS) deficiency has not been established.

“The FDA approval of RAVICTI for children younger than two months provides a new alternative for the management of patients with a UCD that is easy to dose and administer to infants given the liquid formulation,” said Nicola Longo, M.D., Ph.D., clinical geneticist at Primary Children’s Hospital and the University of Utah Hospital, and a lead investigator of a clinical study evaluating RAVICTI for newborns. “UCDs are severe and can be life-threatening. We hope that the combination of early diagnosis – through newborn screening or by measurement of ammonia levels – and the availability of novel treatments, such as this one, can help to improve the outcome of affected patients.”

Click here to read the full press release.