FDA Approves New Drug For The Chronic Management Of Some Urea Cycle Disorders

As a public service announcement, Burke Harvey & Frankowski LLC posts information about new drugs because many of its clients suffer from debilitating conditions.

Hyperion Therapeutics, Inc. announced on Friday, February 1, 2013, that the U.S. Food and Drug Administration (FDA) has approved RAVICTI for the treatment of Urea Cycle Disorders (UCD) in patients two years of age and older.

These inherited conditions involve a lack of enzymes that help the body remove ammonia from the blood. In people with UCDs, ammonia buildup can lead to coma, brain damage or death, the FDA said Friday in a news release.

Ravicti is a nearly tasteless, odorless liquid formulation which is intended to work similarly to Buphenyl in removing ammonia, but without the pill burden or need to mask taste. Three teaspoons of HPN-100 equals approximately 40 tablets of sodium phenylbutyrate. Clinical trials showed improved patient compliance and control of ammonia, including stabilization of fluctuations throughout the day and overnight.  Both children and adults in the trial demonstrated improved cognitive function on Ravicti.

The drug is expected to be commercially available by the end of April 2013.

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