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The LUCASSIN(R) rolling NDA submission is based on the results fromOT-0401,(1) a randomized, double-blind, multi-center, placebo-controlled PhaseIII 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 NDAsubmission.
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"We are pleased to report that, coinciding with the start of our rollingNDA for LUCASSIN(R), the two HRS studies, OT-0401 and TAHRS, have beenpublished 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 tothese two landmark trials, which together represent the largest number of HRSpatients studied in a randomized and controlled study design to-date."
The rolling submission process enables companies that have been grantedfast track designation to submit sections of the NDA to the FDA as they becomeavailable. The FDA grants fast track status to drug candidates that treatserious or life-threatening conditions and that demonstrate the potential toaddress unmet medical needs. Terlipressin received orphan drug designation inOctober 2004 and fast track status in April 2005 for treatment of HRS type 1.
ABOUT HEPATORENAL SYNDROME (HRS)
Hepatorenal syndrome (HRS) is the progressive development of renaldysfunction in patients with late stage liver cirrhosis without any othercauses of renal failure. It is likely caused by a very low blood flow throughthe kidneys, and two types have been described. HRS type 1 is characterized byrapid renal failure with a high mortality rate that exceeds 80% within threemonths. HRS type 2 represents a less aggressive form with longer survival.
The only potentially curative treatment for HRS and its underlying end-stage liver disease is liver transplantation, provided that the patient is asuitable candidate for transplantation and survives until a transplant isavailable. However, many patients may not receive a new liver, furthersupporting the need for alternate therapy options.
ABOUT LUCASSIN(R) (terlipressin)
LUCASSIN(R) (terlipressin) is a synthetic vasopressin analogue that actsvia the vasopressin V1 receptor as a systemic vasoconstrictor, resulting in anincrease in effective arterial volume and improved renal blood flow inpatients with HRS. Terlipressin is not approved by the FDA for use in the U.S.Outside the U.S., terlipressin has been available for over two decades. It hasbeen a standard of care in Europe for esophageal variceal hemorrhage inpatients with liver cirrhosis, and was recently approved in France, Irelandand South Korea for the treatment of patients with HRS type 1.
ABOUT ORPHAN THERAPEUTICS
Orphan Therapeutics, LLC, is a privately held drug development companydedicated to developing treatments for rare and serious diseases. It wasfounded in 2003 with the initial purpose to develop and seek U.S. FDA approvalfor its first product, LUCASSIN(R) (terlipressin), for the treatment ofhepatorenal syndrome (HRS) type 1. For more information contact StephenZoegall, Ph.D., at Berry & Company Public Relations (212 253-8881;szoegall@berrypr.
