Mallinckrodt has announced positive top-line results from its pivotal Phase III CONFIRM clinical study of terlipressin.

The trial evaluated the efficacy and safety of the drug in 300 adults with hepatorenal syndrome type I (HRS-1), and met its primary endpoint of verified HRS-1 reversal, which includes three components: renal function improvement, avoidance of dialysis and short-term survival.

CONFIRM is the largest-ever prospective study (n=300) conducted in patients with HRS-1, an acute and rare disease with high unmet needs and poor prognosis, with no approved therapies in the US or Canada.

"The initial results from the Phase III CONFIRM study are very encouraging in that they demonstrate terlipressin reversed the course of HRS-1 as measured by improvement in renal function, avoidance of dialysis and short-term survival. The study met nearly all of the pre-specified secondary endpoints," said lead investigator Arun Sanyal, Z. Reno Vlahcevic professor of medicine, physiology and molecular pathology at Virginia commonwealth university. "HRS-1 is a life-threatening disease that is extremely difficult to treat. We anticipate the complete results will continue to help inform the effectiveness and safety profile of terlipressin in this patient population with urgent unmet medical needs."

Mallinckrodt has announced plans to submit a New Drug Application to the US Food and Drug Administration (FDA) in early 2020.

HRS-1 is a life-threatening, rare and acute disease characterised by complications of liver disease that lead to kidney failure. HRS-1 has a very poor prognosis, with a median survival time of less than two weeks and greater than 80% mortality within three months.