Europe Clears Ravicti for Urea Cycle Disorders

Megan Brooks

Disclosures

December 01, 2015

The European Commission has approved glycerol phenylbutyrate (Ravicti, Horizon Pharma) as adjunctive therapy for adults and children aged 2 months or older with urea cycle disorder (UCD) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone, the company announced.

The action follows a positive opinion on Ravicti issued by the European Medicines Agency's Committee for Medicinal Products for Human Use in September.

UCD is a rare genetic disorder caused by a mutation that results in a deficiency of one of the six enzymes in the urea cycle. The deficiency ultimately leads to a buildup of ammonia in the blood, which can be fatal.

Glycerol phenylbutyrate, a liquid taken with meals, helps rid the body of ammonia. It must be used with dietary protein restriction and, in some cases, dietary supplements such as essential amino acids, arginine, citrulline, and protein-free calorie supplements.

According to the company, the most common adverse effects are abdominal pain, nausea, diarrhea, and/or headache. The most frequently reported adverse reactions are diarrhea, flatulence, and headache (8.8% each); decreased appetite (7.0%); vomiting (6.1%); and fatigue, nausea, and abnormal skin odor (5.3% each). These reactions usually disappear within a few days, even if treatment is continued.

Ravicti should be prescribed by physicians experienced in the management of UCD, the company said.

The drug was also approved by the US Food and Drug Administration for chronic management of UCD in February 2013.

According to the National Urea Cycle Disorders Foundation, the estimated incidence of UCDs is 1 in 8500 births.

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