Mallinckrodt Plc completed its rolling submission of a NDA to the FDA for terlipressin for the treatment of hepatorenal syndrome type 1.

Mallinckrodt Plc announced the completion of its rolling submission of a New Drug Application (NDA) to the FDA for terlipressin, an investigational agent being evaluated for the treatment of hepatorenal syndrome type 1 (HRS-1). The company previously announced the rolling submission, which it initiated in February. HRS-1 is an acute and life-threatening syndrome involving acute kidney failure in people with cirrhosis. The condition has a median survival time of less than two weeks and greater than 80 percent mortality within three months if left untreated. At present, there are no approved drug therapies for HRS-1 in the U.S., and it is estimated to affect between 30,000 and 40,000 patients in the U.S. annually. In 2005, terlipressin was granted Fast Track designation by the FDA, which provides for expedited review to facilitate development of drugs intended to treat serious or life-threatening conditions and fill an unmet medical nee . In 2016, Mallinckrodt and the FDA reached agreement on the Phase III CONFIRM trial protocol design and data analysis under the agency's special protocol assessment (SPA) process. A SPA is an advance agreement with the FDA for the acceptability of the clinical design, endpoints and statistical data analyses for a Phase III trial before the start of the trial. The submission is a Class 2 resubmission. The terlipressin NDA is based, in part, on results from the Phase III CONFIRM trial, which was the largest-ever prospective study (n=300) conducted in patients with HRS-1, and the culmination of a sustained, 17-year effort to develop terlipressin for potential use in the U.S. and Canada. Initial results were presented at The Liver Meeting 2019, the annual meeting of the American Association For The Study Of Liver Diseases (AASLD).