HZNP announced that the 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, living with a urea cycle disorder (UCD).
We remind investors that Ravicti is already approved by the FDA for use as a nitrogen-binding agent for chronic management of UCDs in adult and pediatric patients older than two months of age with UCDs that cannot be managed by dietary protein restriction and/or amino acid supplementation alone. With the current approval, Ravicti can now be used for chronic management of UCDs in adults and children of all ages.
Ravicti must be used with dietary protein restriction and, in some cases, dietary supplements. Ravicti is not indicated for the 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 current approval provides a new alternative for the the management of patients with UCD that is easy to dose and administer to infants, given that it is a liquid formulation.. The approval was supported by a study, in which 10 patients transitioned to Ravicti from sodium phenylbutyrate, three transitioned from intravenous sodium benzoate and sodium phenylacetate, and three were treatment naive. The study showed safety and efficacy in children younger than two months, with Ravicti-treated patients maintaining stable ammonia levels related to their pre-study enrollment. Further, mean ammonia levels were lower during treatment with Ravicti compared to baseline values.
UCD is a rare genetic disorder that affects approximately 1 in 35,000 live births in the United States.
The drug posted sales of $166.2 million in the first nine months of 2018, an increase of 17% year over year.
Horizon Pharma has another drug, Buphenyl in its portfolio, which is also approved for the treatment of UCD. Both Ravicti and Buphenyl became part of company’s portfolio through the May 2015 Hyperion Therapeutics acquisition.
Shares of Horizon Pharma have increased 27.8% year to date, against the industry’s decline of 26%