Hyperion Therapeutics Receives Orphan Drug Designation for HPN-100 for the Treatment of Urea Cycle...( - Company preparing to initiate phase...),Health

- Company preparing to initiate phase III clinical trial -

SOUTH SAN FRANCISCO, Calif., May 5 /PRNewswire/ -- Hyperion Therapeutics, Inc. today announced that HPN-100 has received orphan product designation from the U.S. Food and Drug Administration for maintenance treatment of patients with enzymes of the urea cycle. The Company recently announced results from its phase II study and intends to initiate a phase III clinical program in this indication in the second half of 2009.

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HPN-100 is a pre-pro-drug of phenylacetic acid which is the active moiety of BUPHENYL(R), the only therapy currently FDA-approved as adjunctive therapy for the chronic management of patients with the most prevalent urea cycle deficiencies including those related to carbamylphosphate synthetase, ornithine transcarbamylase and argininosuccinic acid synthetase. HPN-100 is dosed orally in liquid form.

"Receiving orphan drug designation for HPN-100 in the treatment of urea cycle disorders is an important milestone and provides meaningful benefits to the Company as we work to complete clinical trials for this investigational compound," commented Donald Santel, Chief Executive Officer of Hyperion Therapeutics.

"The designation of HPN-100 as an orphan drug for the treatment of urea cycle disorders recognizes the unmet need that remains in this patient population," said Cindy LeMons, Executive Director of the National Urea Cycle Disorders Foundation (NUCDF). "We are encouraged by this designation and look forward to initiation of the phase III clinical trials."

About Orphan Drug Designation

Orphan drug designation is granted by the FDA Office of Orphan Drug Products to novel drugs or biologics that treat a rare disease or condition
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