US-based biopharmaceutical company Hyperion Therapeutics has announced that it has gained approval from US Food and Drug Administration (FDA) for its RAVICTI (glycerol phenylbutyrate) liquid drug.
RAVICTI used for treating Urea Cycle Disorders (UCD) in patients two years of age and older, is expected to be commercially available by the end of April 2013.
According to the firm, the FDA approval for the new drug was based on a New Drug Application (NDA), which included data from 10 clinical trials, including six in UCD patients.
The trials were conducted at 23 sites, with more than 50 investigators and sub-investigators, and approximately a dozen referring metabolic specialists across the North America.
Hyperion, as part of FDA approval, agreed to fulfil the post-marketing requirements, which include studies of RAVICTI safety, ammonia control, and pharmacokinetics in pediatric UCD patients in the first two months of life and from two months to two years of age.
Hyperion chief medical officer and senior vice president Bruce Scharschmidt said the company believes that the new drug development program has brought not only a promising new medicine to the UCD community, but also a much deeper understanding of UCD treatment.
"The post-marketing studies and registry represent additional opportunities to expand our knowledge in areas that are important to the community, particularly in very young children, a population in which few systematic studies have been done," Scharschmidt added.
