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Horizon's urea disorder drug gets FDA nod for expanded use in newborns

Horizon Pharma PLC's medicine Ravicti was approved by the U.S. Food and Drug Administration to treat infants younger than two months old with a urea cycle disorder.

The Irish biopharmaceutical company said the oral liquid Ravicti, or glycerol phenylbutyrate, is now approved for chronic management of urea cycle disorder, or UCD, in adults and children of all ages whose symptoms cannot be managed by avoiding eating protein or by supplementing with amino acids.

UCD is an inherited disease that makes it hard for a patient's body to remove waste products after digesting proteins. Usually, the nitrogen produced by the breakdown of proteins is converted to a compound called urea and removed from the body, but in UCD the nitrogen accumulates in the form of ammonia. UCD affects about 1 in 35,000 live births in the U.S., said the company.

The company said Ravicti must be used with dietary protein restriction and in some cases with dietary supplements.

Horizon said the approval was based on positive data from a study that showed that children younger than two months who were treated with the drug maintained stable ammonia levels compared to when they had not begun the therapy. Ravicti was also found to be safe in these patients.

Earlier in September, Horizon settled a patent suit against Par Pharmaceutical Inc., which is looking to market a generic version of Ravicti.