STAINES-UPON-THAMES, United Kingdom, March 2, 2020 /PRNewswire/ — Mallinckrodt plc (NYSE: MNK), a global biopharmaceutical company, today announced it initiated the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for terlipressin, an investigational agent being investigated for the treatment of hepatorenal syndrome type 1 (HRS-1), by submitting its clinical data package. A rolling submission allows the company to submit portions of the regulatory application to the FDA as they are completed.1 The company expects to complete the submission of the NDA in the coming months.
HRS-1 is an acute and life-threatening syndrome involving acute kidney failure in people with cirrhosis.2 The condition has a median survival time of less than two weeks and greater than 80 percent mortality within three months if left untreated.3,4 At present, there are no approved drug therapies for HRS-1 in the U.S.,5 and it is estimated to affect between 30,000 and 40,000 patients in the U.S. annually.6,7
“This is an important milestone that brings us closer to potentially delivering the first FDA-approved treatment in the U.S. for HRS-1, a life-threatening, difficult-to-treat condition,” 2 said Steven Romano, M.D., Executive Vice President and Chief Scientific Officer at Mallinckrodt. “We are grateful to the patients, their families and caregivers, and the researchers who made this achievement possible.”
The terlipressin NDA is based, in part, on results from the Phase 3 CONFIRM trial, which was the largest-ever prospective study (n=300) conducted in patients with HRS-1, and the culmination of a sustained, 17-year effort to develop terlipressin for potential use in the U.S. and Canada. The CONFIRM trial met its primary endpoint of Verified HRS Reversal (VHRSR), defined as renal function improvement, avoidance of dialysis and short-term survival (p=0.012). Serious adverse events were reported in 65 percent (n=130) of subjects in the terlipressin group and 60.6 percent (n=60) in the placebo group. No new or unexpected AEs were reported. Initial results were presented at The Liver Meeting® 2019, the annual meeting of the American Association for the Study of Liver Diseases (AASLD).
Terlipressin is a potent vasopressin analogue selective for V1 receptors being investigated for the treatment of HRS-1 in the U.S. and Canada. It is an investigational product in these countries as the safety and efficacy have not been established with, nor has approval been granted by, regulatory authorities in either country. Terlipressin is approved for use outside the U.S. and Canada.
Mallinckrodt is a global business consisting of multiple wholly owned subsidiaries that develop, manufacture, market and distribute specialty pharmaceutical products and therapies. The company’s Specialty Brands reportable segment’s areas of focus include autoimmune and rare diseases in specialty areas like neurology, rheumatology, nephrology, pulmonology and ophthalmology; immunotherapy and neonatal respiratory critical care therapies; analgesics and gastrointestinal products. Its Specialty Generics reportable segment includes specialty generic drugs and active pharmaceutical ingredients. To learn more about Mallinckrodt, visit www.mallinckrodt.com.
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CAUTIONARY STATEMENTS RELATED TO FORWARD-LOOKING STATEMENTS
This release includes forward-looking statements with regard to terlipressin, including the expected timing for completion of Mallinckrodt’s submission to the FDA and its potential impact on patients. The statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those in the forward-looking statements: satisfaction of regulatory and other requirements; actions of regulatory bodies and other governmental authorities; changes in laws and regulations; issues with product quality, manufacturing or supply, or patient safety issues; and other risks identified and described in more detail in the “Risk Factors” section of Mallinckrodt’s most recent Annual Report on Form 10-K and other filings with the SEC, all of which are available on its website. The forward-looking statements made herein speak only as of the date hereof and Mallinckrodt does not assume any obligation to update or revise any forward-looking statement, whether as a result of new information, future events and developments or otherwise, except as required by law.
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1 U.S. Food and Drug Administration. “Guidance for Industry Expedited Programs for Serious Conditions – Drugs and Biologics.” Available at https://www.fda.gov/files/drugs/published/Expedited-Programs-for-Serious-Conditions-Drugs-and-Biologics.pdf. Accessed February 6, 2020.
2 National Organization for Rare Disorders. Hepatorenal Syndrome. Available at: https://rarediseases.org/rare-diseases/hepatorenal-syndrome/. Accessed April 9, 2019.
3 Colle I and Laterre PF. Hepatorenal syndrome: the clinical impact of vasoactive therapy, Expert Review of Gastroenterology & Hepatology. (2018) 12:2, 173-188, DOI: 10.1080/17474124.2018.1417034.
4 Gines P, Sola E, Angeli P, et al. Hepatorenal syndrome. Nature Reviews. (2018) 4:23.
5 Boyer TD, Medicis JJ, Pappas SC, et al. A randomized, placebo-controlled, double-blind study to confirm the reversal of hepatorenal syndrome type 1 with terlipressin: the REVERSE trial design. Open Access Journal of Clinical Trials 2012:4. https://www.dovepress.com/a-randomized-placebo-controlled-double-blind-study-to-confirm-the-reve-peer-reviewed-article-OAJCT.
6 C Pant, B S Jani, M Desai, A Deshpande, Prashant Pandya, Ryan Taylor, R Gilroy, M Olyaee. Hepatorenal syndrome in hospitalized patients with chronic liver disease: results from the Nationwide Inpatient Sample 2002–2012. J Investig Med 2016;64:33–38.
7 US Census 2018 https://www.census.gov/search-results.html?searchType=web&cssp=SERP&q=US population 2018, accessed on 06 August 2019.