Orphan Therapeutics Announces Initiation of Rolling NDA Submission for LUCASSIN(R) (terlipressin) for the Treatment of Hepatorenal Syndr...( LEBANON N.J. May 28 /- Orphan The...),Health

LEBANON, N.J., May 28 /PRNewswire/ -- Orphan Therapeutics today announced that it has begun submission of the rolling LUCASSIN(R) (terlipressin) New Drug Application (NDA) for the treatment of hepatorenal syndrome (HRS) type 1 in patients with late-stage liver cirrhosis. LUCASSIN(R) had previously been granted orphan status and fast track designation for this indication by the U.S. Food and Drug Administration (FDA). Currently no drug is approved in the U.S. to treat HRS type 1, a rare and life-threatening condition in late-stage liver disease.

The LUCASSIN(R) rolling NDA submission is based on the results from OT-0401,(1) a randomized, double-blind, multi-center, placebo-controlled Phase III study in 112 patients with HRS type 1 conducted by Orphan Therapeutics, and is independently supported by TAHRS,(2) a second randomized, multi-center, controlled study in 46 patients coordinated by the University of Barcelona. Orphan Therapeutics holds exclusive rights to the TAHRS data for the NDA submission.

"We are pleased to report that, coinciding with the start of our rolling NDA for LUCASSIN(R), the two HRS studies, OT-0401 and TAHRS, have been published in the May 2008 issue of Gastroenterology," said Peter Teuber, president of Orphan Therapeutics. "I would like to thank all authors, investigators and members of the LUCASSIN(R) team for their contributions to these two landmark trials, which together represent the largest number of HRS patients studied in a randomized and controlled study design to-date."

The rolling submission process enables companies that have been granted fast track designation to submit sections of the NDA to the FDA as they become available. The FDA grants fast track status to drug candidates that treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs. Terlipressin received orphan drug designation in October 2004 and fast track status in April 2005 for treatment of
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