With the submission of its clinical data package, UK biopharma, Mallinckrodt, has initiated the rolling submission of a New Drug Application (NDA) to US regulators for terlipressin, an investigational agent being investigated for the treatment of hepatorenal syndrome type 1 (HRS-1).
A rolling submission allows the company to submit portions of the regulatory application to the FDA as they are completed. The company expects to complete the submission of the NDA in the coming months.
If approved, terlipressin would be the first and only FDA-approved treatment option in the US for patients with HRS-1, an acute and life-threatening syndrome involving acute kidney failure in people with cirrhosis.
The NDA is based, in part, on results from the Phase 3 CONFIRM trial, which was the largest-ever prospective study conducted in patients with HRS-1, and the culmination of a sustained, 17-year effort to develop terlipressin for potential use in the US and Canada.
The trial met its primary endpoint of Verified HRS Reversal, defined as renal function improvement, avoidance of dialysis and short-term survival.