Horizon files sNDA to extend the indication of Ravicti to include infants younger than two months of age living with urea cycle disorders.

Horizon Pharma plc announced that it has submitted a supplemental New Drug Application (sNDA) with the FDA to expand the approved indication for Ravicti (glycerol phenylbutyrate) Oral Liquid to include infants younger than two months of age living with urea cycle disorders (UCDs).

Currently,Ravicti is indicated for use as a nitrogen-binding agent for chronic management of adult and pediatric patients greater than two months of age with UCDs who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. Ravicti is not indicated for treatment of acute hyperammonemia in patients with UCDs, and the safety and efficacy of Ravicti for the treatment of n-acetylglutamate synthase (NAGS) deficiency has not been established. The sNDA seeks to expand Ravicti�s indication to include an age range down to birth, and is based on an open-label clinical study evaluating 16 patients with UCDs less than two months of age. The FDA is expected to complete their review during the second half of 2018.

�Urea cycle disorders are typically most severe when the onset of symptoms begin early in life, and symptoms that begin right after birth are particularly challenging,� said Shao-Lee Lin, M.D., Ph.D., executive vice president, head of research and development and chief scientific officer, Horizon Pharma plc. �As part of Horizon�s ongoing effort to reinvest in our medicines, this sNDA submission brings us closer to achieving our mission to make Ravicti available for the majority of those living with a UCD, including newborns.�