Horizon Pharma has announced that Ravicti (glycerol phenylbutyrate) Oral Liquid is now available in Canada through its affiliate Horizon Therapeutics Canada Limited.
Horizon noted in its press release issued on Thursday that in March 2016 it had received a Notice of Compliance (NOC) from Health Canada for Ravicti for use as an adjunctive therapy for chronic management of adult and pediatric patients two years of age and older with Urea Cycle Disorders (UCDs).
“The availability of Ravicti provides an important new treatment option for Canadians living with UCDs,” said Jared Rhines, vice president and general manager, Canada, LATAM and APAC, Horizon Pharma. “Through our participation in UCD education and awareness programs around the world, our team has been inspired by the strength and resilience of people living with this rare and potentially devastating condition. This interaction drives our commitment to support and partner with the UCD community, including the healthcare professionals, families and caregivers of people living with UCDs in Canada and beyond.”
UCDs are inherited metabolic diseases caused by a deficiency of one of the enzymes or transporters that constitute the urea cycle. The urea cycle involves a series of biochemical steps in which ammonia, a potent neurotoxin, is converted to urea, which is excreted in the urine. UCD patients may experience episodes as a result of elevated ammonia levels in their blood – called hyperammonemic crises – which may result in 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.
“We have heard from so many patients and families about their challenges trying to manage this very rare and devastating condition. We have heard over and over about the struggles to get a diagnosis and the feelings of isolation,” said Durhane Wong-Rieger, president and chief executive officer, Canadian Organization for Rare Disorders (CORD). “Having new treatment options for those living with UCDs in Canada is important, and one of our top priorities at CORD is to help ensure that people living with rare diseases have access to innovative medicines.”