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 U.S. Food and Drug Administration (FDA) has approved its supplemental New Drug Application (sNDA) to expand the age range for RAVICTI® (glycerol phenylbutyrate) Oral Liquid to people two months of age and older who have urea cycle disorders (UCDs) that cannot be managed by dietary protein restriction and/or amino acid supplementation alone. RAVICTI initially was approved by the FDA in 2013 for adults and children as young as two years of age.
“Through our ongoing collaboration with physicians and parents of children living with UCDs, we understand the devastating effects of hyperammonemic events early in a child’s life,” said Jeffrey W. Sherman, M.D., FACP, executive vice president, research and development and chief medical officer, Horizon Pharma plc. “The approval by the FDA of our sNDA to expand RAVICTI to children as young as two months is an important step in helping young children with UCDs. We will continue to invest in RAVICTI to address the unmet needs of those with UCDs, as well as help their caregivers and family members more confidently tackle the challenges of the disease.”
The approval is based on three studies that assessed monthly ammonia control and hyperammonemic crises (HACs) in pediatric patients with UCDs two months to two years of age. Patients were treated with RAVICTI for an average of eight months, and received RAVICTI either at study initiation or by enrolling on stable doses of sodium phenylbutyrate or sodium benzoate then switching to equivalent doses of RAVICTI. Results found RAVICTI to be safe and effective in the pediatric patients studied, with RAVICTI-treated patients maintaining stable ammonia levels relative to their pre-study enrollment.
“This approval represents a significant advance for very young children with UCDs, one of the most vulnerable patient populations, as more severe cases of the disease tend to present earlier in life and can lead to serious long-term impairments if not diagnosed and treated early,” said Susan Berry, M.D., professor and division director for genetics and metabolism, department of pediatrics, University of Minnesota and primary investigator for the RAVICTI studies leading to the sNDA approval.
A UCD is a rare genetic disorder that affects approximately 1 in 35,000 live births in the United States. It is caused by an enzyme deficiency in the urea cycle, a process that is responsible for converting excess ammonia from the bloodstream and ultimately removing it from the body. Because of this, people with a UCD experience hyperammonemia, or elevated ammonia levels in their blood, that can then reach the brain and cause irreversible brain damage, coma or death. UCD symptoms may first occur at any age depending on the severity of the disorder, with more severe defects presenting earlier in life.
RAVICTI was first approved in the U.S. in February 2013 for the chronic management of adult and pediatric patients ≥2 years of age with UCDs that cannot be managed by dietary protein restriction and/or amino acid supplementation alone. In April 2017, the indication for RAVICTI was expanded to include children as young as two months of age. Click here for more information about RAVICTI.