The FDA has approved Ravicti (glycerol phenylbutyrate; Hyperion) liquid for the chronic management of some urea cycle disorders in patients aged >2 years who cannot be managed by a protein-restricted diet and/or amino acid supplements alone.

Ravicti, formerly known as HPN-100, is a pre-pro-drug of phenylacetic acid, the active moiety of Buphenyl (sodium phenylbutyrate; Ucyclyd Pharma). It helps dispose of ammonia in the body and must be used while on a protein-restricted diet with or without dietary supplements (eg, essential amino acids, arginine, citrulline, protein-free calorie supplements).

The safety and efficacy of Ravicti was based on a New Drug Application (NDA) that included data from 10 clinical trials, including six in urea cycle disorder patients that involved 23 sites, over 50 investigators and sub-investigators, and approximately a dozen referring metabolic specialists throughout North America. Post-marketing studies required by the FDA include studies on safety, ammonia control, drug interactions, a 10-year registry, and more.

Ravicti will be available in 1.1g/mL liquid in 25mL multi-use glass vials. It is expected to be available by the end of April 2013.

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