Mallinckrodt Receives a Complete Response Letter from the U.S. Food and Drug Administration (FDA) for Terlipressin for the Treatment of Hepatorenal Syndrome Type 1 (HRS-1)
Mallinckrodt Receives a Complete Response Letter from the U.S. Food and Drug Administration (FDA) for Terlipressin for the Treatment of Hepatorenal Syndrome Type 1 (HRS-1) Company remains committed to working with FDA toward an approval for this important potential therapy |
| [14-September-2020] |
DUBLIN, Sept. 14, 2020 /PRNewswire/ -- Mallinckrodt plc (NYSE: MNK), a global biopharmaceutical company, today announced that the U.S. Food and Drug Administration (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 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 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.7 "While we are disappointed that the FDA issued a complete response letter for terlipressin, we remain confident in the strength of the data from our Phase 3 CONFIRM study, which is the largest clinical trial ever conducted in this rare condition," said Steven Romano, M.D., Executive Vice President and Chief Scientific Officer at Mallinckrodt. "HRS-1 is a complex disease that affects a critically ill patient population with no approved treatment in the U.S. at present. We are surprised by and disagree with the FDA's decision and remain committed to pursuing all available options as we continue working with the FDA toward approval of terlipressin in order to help address this difficult and life-threatening syndrome." Terlipressin is an investigational agent being evaluated for the treatment of HRS-1 in the U.S., and its safety and effectiveness have not yet been established by the FDA. Terlipressin is approved in many countries outside the United States, where it has been a standard of care for decades in the treatment of patients with HRS-1.8,9 Terlipressin, together with albumin, is currently the standard of care for HRS-1 in countries where it is available.10 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.11 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. 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 3 CONFIRM trial. The CONFIRM trial was the largest-ever prospective study (n=300) conducted to assess the safety and efficacy of terlipressin in patients with HRS-1 for potential use in the U.S. and Canada. Initial results were presented in a late-breaking session at The Liver Meeting® 2019, the annual meeting of the American Association for the Study of Liver Diseases (AASLD). About Terlipressin ABOUT MALLINCKRODT 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 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-2001369 09/20 References 1 National Organization for Rare Disorders. Hepatorenal Syndrome. Available at: https://rarediseases.org/rare-diseases/hepatorenal-syndrome/. Accessed September 12, 2020.
SOURCE Mallinckrodt Pharmaceuticals | ||
Company Codes: NYSE:MNK |
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