Initial results from a phase 3 clinical study of terlipressin showed that the therapy met its primary endpoint of “verified” hepatorenal syndrome type 1 reversal, according to a press release from Mallinckrodt.
“The initial results from the phase 3 CONFIRM study are very encouraging in that they demonstrate terlipressin reversed the course of [hepatorenal syndrome type 1 (HRS-1)] as measured by improvement in renal function, avoidance of dialysis and short-term survival,” Arun Sanyal, MD, from the Virginia Commonwealth University, said in the release. “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.”
Terlipressin is an analogue of vasopressin that is selective for V1 receptors under investigation for HRS-1.
The randomized control study comprised 300 patients with HRS-1, cirrhosis and ascites, and rapidly worsening renal function with no response to diuretics or albumin. Patients demonstrated all endpoints for HRS reversal (P = .012).
“At Mallinckrodt, we are committed to advancing therapies that treat underserved patients with severe and critical conditions, including patients with this devastating condition,” Steven Romano, MD, executive vice president and chief scientific officer at Mallinckrodt, said in the release. “The CONFIRM trial will support regulatory submissions of terlipressin as a treatment for HRS-1 in the U.S. and Canada, and we look forward to sharing full results soon.”
The company plans to submit a new drug application to the FDA in early 2020.