Hyperion Confirms FDA Approval for RAVICITI
Hyperion Therapeutics, Inc. (Nasdaq: HPTX) today announced 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. The drug is expected to be commercially available by the end of April 2013.
Separately, Hyperion announced it has received notification from the U.S. Patent & Trademark Office (USPTO) of the allowance of the claims of patent application number 13/417,137 entitled, METHODS OF THERAPEUTIC MONITORING OF NITROGEN SCAVENGING DRUGS, which discloses optimal measurement timing and target levels for blood ammonia in UCD patients. A Notice of Allowance is issued after the USPTO makes a determination that a patent can be granted from an application. The issued patent would have a term that expires in March 2032. After issuance, Hyperion plans to list this patent in FDA's Approved Drug Products with Therapeutic Equivalence, or Orange Book.
"With FDA approval of RAVICTI, we are now in a position to commercialize Hyperion's first product. Furthermore we believe that the allowed claims, once issued, will provide important protection for the use of RAVICTI to treat UCD," said Donald J. Santel, Hyperion's chief executive officer. "We thank our clinical trial patients, their families, our investigators and study coordinators, the UCD Consortium, and the National Urea Cycle Disorders Foundation – in particular NUCDF Executive Director Cynthia Le Mons – for their collaboration and support of our development efforts."
The FDA approval of RAVICTI was based on a New Drug Application (NDA) that included data from 10 clinical trials, including six in UCD patients, that involved 23 sites, over 50 investigators and sub-investigators, and approximately a dozen referring metabolic specialists throughout North America. As part of FDA approval, Hyperion agreed to post-marketing requirements that 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; studies in healthy adults to examine drug-drug interaction and whether RAVICTI metabolites are present in breast milk; a randomized controlled clinical trial to assess the safety and efficacy of RAVICTI in treatment-naïve patients with UCD; and a UCD registry of approximately 10 years duration. Importantly, the FDA did not require a Risk Evaluation and Mitigation Strategy (REMS) program.
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