The Food and Drug Administration has approved Horizon Pharma's supplemental new drug application to expand the use of Ravicti (glycerol henylbutyrate), an oral liquid used to treat urea cycle disorders (UCDs) in patients who are unable to manage the condition with dietary restriction or amino acid supplementation, to patients as young as 2 months old.
Ravicti had previously been approved for patients 2 years old and older, Horizon Pharma said in a release.
Ravicti (glycerol henylbutyrate) is an oral liquid used to treat urea cycle disorders (UCDs) in patients who are unable to manage the condition with dietary restriction or amino acid supplementation.
"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," Susan Berry, professor and division director for genetics and metabolism, department of pediatrics, University of Minnesota, was quoted as saying in the release. Berry was the primary investigator for the Ravicti studies leading to the approval.
There were three studies done that looked at monthly ammonia control and hyperamonemic crises in young patients. Researchers treated patients with Ravicti and found the medicine to be not only safe for pediatric patients as young as 2 months old but also a reliable way to stabilize their ammonia levels, the release said.
UCD is caused by a deficient enzyme in the urea cycle, which can affect the ammonia levels in the blood of those with the condition. There is a risk that these high levels of ammonia can cause brain damage, coma or even death, the release said. It affects approximately 1 in 35,000 babies born in the United States.
