Mallinckrodt Announces U.S. Food and Drug Administration (FDA) Filing Acceptance of New Drug Application for Terlipressin for Treatment of Hepatorenal Syndrome Type 1 (HRS-1)
Mallinckrodt Announces U.S. Food and Drug Administration (FDA) Filing Acceptance of New Drug Application for Terlipressin for Treatment of Hepatorenal Syndrome Type 1 (HRS-1) - Terlipressin would be the first FDA-approved treatment option in the United States for adult patients with HRS-1, a life-threatening condition, if approved - |
[22-April-2020] |
STAINES-UPON-THAMES, United Kingdom, April 22, 2020 /PRNewswire/ -- Mallinckrodt plc (NYSE: MNK), a global biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the company's New Drug Application (NDA) for terlipressin, an investigational agent being evaluated for the treatment of hepatorenal syndrome type 1 (HRS-1). On March 17, 2020 the company announced the completion of its rolling submission of the NDA for terlipressin. The FDA assigned a Prescription Drug User Fee Act (PDUFA) target date of September 12, 2020. HRS-1 is an acute and life-threatening syndrome involving acute kidney failure in people with cirrhosis.1 The condition has a median survival time of approximately two weeks and greater than 80 percent mortality within three months if left untreated.2,3 At present, there are no approved drug therapies for HRS-1 in the U.S.,4 and it is estimated to affect between 30,000 and 40,000 patients in the U.S. annually.5,6 "We are pleased that we received filing acceptance of our NDA submission. This is an important milestone in our clinical development program for terlipressin to address a critically high unmet need for patients with HRS-1 and their physicians, who historically have had limited treatment options," said Steven Romano, M.D., Executive Vice President and Chief Scientific Officer at Mallinckrodt. "We look forward to working with the agency during their review of our regulatory package." In 2005, terlipressin was granted Fast Track designation by the FDA, which provides for expedited review to facilitate development of drugs intended to treat serious or life-threatening conditions and fill an unmet medical need.7 In 2016, Mallinckrodt and the FDA reached agreement on the Phase 3 CONFIRM trial protocol design and data analysis under the agency's special protocol assessment (SPA) process. A SPA is an advance agreement with the FDA for the acceptability of the clinical design, endpoints and statistical data analyses for a Phase 3 trial before the start of the trial. The submission is a Class 2 resubmission. Mallinckrodt anticipates an FDA Advisory Committee meeting to discuss the submission later this year. 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. Initial results were presented at The Liver Meeting® 2019, the annual meeting of the American Association for the Study of Liver Diseases (AASLD). About Terlipressin ABOUT MALLINCKRODT 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. Mallinckrodt uses its website as a channel of distribution of important company information, such as press releases, investor presentations and other financial information. It also uses its website to expedite public access to time-critical information regarding the company in advance of or in lieu of distributing a press release or a filing with the U.S. Securities and Exchange Commission (SEC) disclosing the same information. Therefore, investors should look to the Investor Relations page of the website for important and time-critical information. Visitors to the website can also register to receive automatic e-mail and other notifications alerting them when new information is made available on the Investor Relations page of the website. CAUTIONARY STATEMENTS RELATED TO FORWARD-LOOKING STATEMENTS CONTACT For Trade Media Inquiries For Financial/Dailies Media Inquiries Investor Relations 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-2000713 04/20 References 1 National Organization for Rare Disorders. Hepatorenal Syndrome. Available at: https://rarediseases.org/rare-diseases/hepatorenal-syndrome/. Accessed April 20, 2020.
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