Variquel starts rolling submission process with FDA for hepatorenal syndrome type 1.- Mallinckrodt

Mallinckrodt announced it initiated the rolling submission of a New Drug Application (NDA) to the FDA for Variquel (terlipressin), an investigational agent being investigated for the treatment of hepatorenal syndrome type 1 (HRS-1), by submitting its clinical data package. The company expects to complete the submission of the NDA in the coming months.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. The CONFIRM trial met its primary endpoint of Verified HRS Reversal (VHRSR), defined as renal function improvement, avoidance of dialysis and short-term survival (p=0.012). Serious adverse events were reported in 65 percent (n=130) of subjects in the terlipressin group and 60.6 percent (n=60) in the placebo group. No new or unexpected AEs were reported. Initial results were presented at The Liver Meeting 2019.