Complete Response Letter for terlipressin to treat hepatorenal syndrome type 1.- Mallinckrodt

Mallinckrodt Plc announced that the FDA has issued a Complete Response Letter (CRL) regarding the company's New Drug Application (NDA) seeking approval for the investigational agent terlipressin to treat adults with hepatorenal syndrome type 1 (HRS-1). The CRL stated that, based on the available data, the agency cannot approve the terlipressin NDA in its current form and requires more information to support a positive risk-benefit profile for terlipressin for patients with HRS-1. HRS-1 is an acute and life-threatening syndrome involving acute kidney failure in people with cirrhosis for which there is currently no FDA-approved treatment. HRS-1 is estimated to affect between 30,000 and 40,000 Americans annually and often is a challenge to effectively diagnose in a timely manner due to its diagnosis of exclusion. If left untreated, HRS-1 has a median survival time of approximately two weeks and greater than 80 percent mortality within three months. U.S. discharge data in a recently published study indicated an in-hospital mortality rate of 34.2% (n=1,133), while an additional 14.4% (n=475) of patients were discharged to hospice. On July 15, 2020, the company announced that the Cardiovascular And Renal Drugs Advisory Committee Of the FDA voted to recommend approval of its investigational agent terlipressin to treat adults with HRS-1 based, in part, on results from the Phase III CONFIRM trial.