BRIEF—Ravicti now available for all ages in Europe

Swedish Orphan Biovitrum (Sobi) announces that Ravicti (glycerol phenylbutyrate), a therapy option for the treatment of patients with urea cycle disorders (UCDs), had received approval from the European Commission on December 18, 2018 for the pediatric indication (birth to two months of age) following a positive opinion from the European Medicines Agency advisory committee, CHMP.

The previous indication for Ravicti was restricted to children aged more than two months.

“This new expanded indication allows all patients access to treatment, which is particularly significant as the disease often presents in newborns,” says Norbert Oppitz, head of

“Following stabilization with acute intravenous therapy, children can now be managed long-term on Ravicti in the early days of their lives,” he added.

The approval was based on a multicenter, open-label clinical study of the safety, efficacy and pharmacokinetics of glycerol phenylbutyrate in pediatric subjects under two years of age with UCDs, carried out by Horizon Pharma.

The study, which included a cohort of 16 patients from birth to two months, demonstrated that glycerol phenylbutyrate is safe and effective in establishing and maintaining ammonia control in pediatric UCD subjects aged from birth to two years.