DUBLIN – November 13, 2020 – Mallinckrodt plc, a global biopharmaceutical company, today announced that the Company recently participated in an end of review meeting with the U.S. Food and Drug Administration (FDA) to discuss the Complete Response Letter issued on September 11, 2020 for the Company’s New Drug Application (NDA) for its investigational agent terlipressin to treat adults with hepatorenal syndrome type 1 (HRS-1).
Based on the discussion during an end of review meeting, the Company plans to continue engaging with the FDA to explore options for a potential regulatory path forward regarding the terlipressin NDA. Mallinckrodt plans to request a meeting with the FDA in Q1 2021 to formally discuss possible options to move the terlipressin development program forward.
“We are encouraged by our ongoing dialogue with the FDA and look forward to meeting with the agency again in the first quarter of next year,” said Steven Romano, M.D., Executive Vice President and Chief Scientific Officer at Mallinckrodt. “We remain steadfast in our commitment to work toward bringing a new HRS-1 treatment option to patients in the U.S. to help address this difficult and life-threatening syndrome."
HRS-1 is an acute and life-threatening syndrome involving acute kidney failure in people with cirrhosis1 for which there is currently no FDA-approved treatment.2 HRS-1 is estimated to affect between 30,000 and 40,000 Americans annually,3,4 and often is a challenge to effectively diagnose in a timely manner due to its diagnosis of exclusion.5 If left untreated, HRS-1 has a median survival time of approximately two weeks and greater than 80 percent mortality within three months.5,6
Terlipressin is an investigational product and its safety and effectiveness have not yet been established by the U.S. FDA.
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, hepatology, 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, with regard to interactions with regulators as well as 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.
Green Room Communications
Daniel J. Speciale
Vice President, Finance and Investor Relations Officer
Mallinckrodt, the "M" brand mark and the Mallinckrodt Pharmaceuticals logo are trademarks of a Mallinckrodt company. Other brands are trademarks of a Mallinckrodt company or their respective owners. © 2020 Mallinckrodt. US-2001838 11/20
1 National Organization for Rare Disorders. Hepatorenal Syndrome. Available at: https://rarediseases.org/rare-diseases/hepatorenal-syndrome/. Accessed November 9, 2020.
2 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.
3 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. Journal of Investigative Medicine. 2016; 64:33–38.
4 United States Census Bureau: Quick Facts. Available at: https://www.census.gov/quickfacts/fact/table/US/PST045218. Accessed November 9, 2020.
5 Gines P, Sola E, Angeli P, et al. Hepatorenal syndrome. Nature Reviews. 2018;4:23.
6 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.