{"id":450384,"date":"2021-03-04T06:48:36","date_gmt":"2021-03-04T11:48:36","guid":{"rendered":"http:\/\/www.marketnewsdesk.com\/?p=450384"},"modified":"2021-03-04T06:48:36","modified_gmt":"2021-03-04T11:48:36","slug":"mallinckrodt-announces-new-england-journal-of-medicine-publication-of-results-from-its-phase-3-confirm-study-of-terlipressin-in-patients-with-hepatorenal-syndrome-type-1-hrs-1","status":"publish","type":"post","link":"https:\/\/www.marketnewsdesk.com\/index.php\/mallinckrodt-announces-new-england-journal-of-medicine-publication-of-results-from-its-phase-3-confirm-study-of-terlipressin-in-patients-with-hepatorenal-syndrome-type-1-hrs-1\/","title":{"rendered":"Mallinckrodt Announces New England Journal of Medicine Publication of Results from its Phase 3 CONFIRM Study of Terlipressin in Patients with Hepatorenal Syndrome Type 1 (HRS-1)"},"content":{"rendered":"
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— CONFIRM is the largest prospective study (n=300) conducted in patients with HRS-1, a life-threatening and acute form of advanced liver disease with high unmet needs and a poor prognosis —<\/h2>\n

PR Newswire<\/p>\n<\/p><\/div>\n

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\n DUBLIN<\/span>, March 4, 2021<\/span> \/PRNewswire\/ — Mallinckrodt<\/span> plc<\/a>, a global biopharmaceutical company, today announced publication of results from its pivotal Phase 3 CONFIRM study to assess the efficacy and safety of its investigational agent terlipressin in adults with hepatorenal syndrome type 1 (HRS-1). HRS-1 is an acute and life-threatening syndrome involving acute kidney failure in patients with cirrhosis,1<\/sup> and has a median survival time of approximately two weeks and greater than 80 percent mortality within three months if left untreated.2<\/sup><\/sup>,3<\/sup><\/sup>\u00a0The study was posted online<\/a> ahead of print publication in the New England Journal of Medicine.<\/i><\/p>\n

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Terlipressin is an investigational product and its safety and effectiveness have not yet been established by the U.S. Food and Drug Administration (FDA) or Health Canada.<\/p>\n

As previously announced, the Phase 3 CONFIRM study met its primary endpoint of Verified HRS Reversal, which is defined as renal function improvement, avoidance of dialysis and short-term survival. The main objective of the CONFIRM study was to assess the efficacy and safety of terlipressin, together with albumin, versus placebo in adults in the U.S. and Canada<\/span> with cirrhosis and HRS-1. The trial met three of the four pre-specified secondary endpoints of the study including HRS reversal,\u00a0<\/b>HRS reversal without renal replacement therapy (RRT) by Day 30 and HRS reversal in the systemic inflammatory response syndrome (SIRS) subgroup. The fourth pre-specified secondary endpoint of Verified HRS Reversal without HRS recurrence by Day 30 was 50 percent greater in the terlipressin group but did not reach statistical significance.4<\/sup> Initial results were announced<\/a> during a late-breaking abstract presentation on November 11, 2019<\/span> at The Liver Meeting\u00ae<\/sup>, the annual meeting of the American Association for the Study of Liver Diseases (AASLD).<\/p>\n

In another pre-specified endpoint, avoidance of RRT, terlipressin treated subjects (n=199) showed a significantly lower incidence during the treatment period and a lower incidence at all follow-up assessments through Day 90 versus patients with placebo (n=101).4\u00a0This is clinically significant because RRT can increase complications in HRS-1 due to the underlying cirrhosis (i.e. coagulopathy, low blood pressure).\u00a0\u00a0<\/p>\n

“The durability of HRS reversal with terlipressin in CONFIRM persisted to Day 30 without the need for RRT. This is a clinically significant observation, as RRT poses many challenges for patients with advanced cirrhosis,” said lead author Florence Wong<\/span>, MBBS, MD, FRACP, FRCPC, Hepatologist at Toronto<\/span> General Hospital and Professor of Medicine at the University of Toronto<\/span><\/b>. “Results from CONFIRM provide critical information on a potential treatment option for HRS-1 and these data indicate that, if approved, terlipressin has the potential to reverse the course of HRS-1 in the appropriate patients and help the healthcare community better manage this critically ill and underserved patient population.”<\/p>\n

The incidence of adverse events (AEs) of any severity were similar in both groups (88.0 percent of the terlipressin group and 88.9 percent of the placebo group). The most commonly reported AEs in the overall study population were abdominal pain, nausea, diarrhea, hepatic encephalopathy and dyspnea. Serious AEs (SAEs) were reported in 65.0 percent (n=130) of the terlipressin group and 60.6 percent (n=60) of the placebo group. The most commonly reported SAEs included hepatobiliary disorders, respiratory disorders and gastrointestinal disorders.4<\/sup>\u00a0<\/p>\n

At present, there are no drug therapies approved for the treatment of HRS-1 in the U.S. or\u00a0Canada.5<\/sup>\u00a0HRS-1 is estimated to affect between 30,000 and 40,000 patients in the U.S.\u00a0annually.6,7<\/sup><\/p>\n

In November 2020<\/span>, Mallinckrodt<\/span>announced<\/a> it participated in an end of review meeting with the FDA to discuss the Complete Response Letter issued on September 11, 2020<\/span> for the Company’s New Drug Application (NDA) for terlipressin. Based on recent discussions with the FDA, Mallinckrodt<\/span> continues to explore a potential regulatory path forward regarding the NDA.\u00a0<\/p>\n

Terlipressin is approved in many countries outside the U.S. and Canada<\/span>, where it has been a standard of care for decades in the treatment of patients with HRS-1.8,9<\/sup>\u00a0Terlipressin, together with albumin, is currently the standard of care for HRS-1 in countries where it is approved and available.10<\/sup><\/sup><\/p>\n

“The CONFIRM results provide meaningful insight into the management of HRS-1 in clinical practice, and we are pleased to be able to share these important data broadly with the healthcare community,” said Steven Romano<\/span>, M.D., Executive Vice President and Chief Scientific Officer at\u00a0Mallinckrodt<\/b>. “On behalf of Mallinckrodt<\/span>, I would also like to once again thank all of the patients, caregivers and medical professionals whose contributions made this study possible.”\u00a0\u00a0<\/p>\n

\n About the Pivotal Phase 3 CONFIRM Study (multi-center, randomized, placebo-controlled, double-blind trial in the U.S. and Canada<\/span>)<\/b>
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