The U.S. Food and Drug Administration approved Hyperion Therapeutics Inc.'s (HPTX) Ravicti or glycerol phenylbutyrate for the chronic management of some urea cycle disorders or UCDs in patients ages 2 years and older.
UCDs are genetic disorders that involve deficiencies of specific enzymes involved in the urea cycle, a series of biochemical steps normally required to remove ammonia from the blood. When protein is absorbed and broken down by the body, it produces nitrogen as a waste product.
The urea cycle removes nitrogen from the blood and converts it to urea, which is removed from the body through urine. In people with UCDs, nitrogen accumulates and remains in the body as ammonia, which can travel to the brain and cause brain damage, coma or death.
Ravicti, a liquid taken three times a day with meals, helps dispose of ammonia in the body. It is intended for patients whose UCD cannot be managed by a protein-restricted diet or amino acid supplements alone. Ravicti must be used with a protein-restricted diet and, in some cases, dietary supplements.
Ravicti was reviewed under the agency's fast track program, designed to facilitate the development and expedite the review of drugs to treat serious diseases, fill unmet medical needs, and get important new drugs to patients earlier.
Ravicti also was granted orphan product designation because it is intended to treat a rare disease.
According to the FDA, the major study supporting Ravicti's safety and effectiveness involved 44 adults who had been using Buphenyl, another drug approved to control UCDs.
The most common side effects in patients treated with Ravicti include diarrhea, flatulence and headache.
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