Mallinckrodt Confirms that U.S. Food and Drug Administration (FDA) Will Convene an Advisory Committee to Review Terlipressin for the Treatment of Patients with Hepatorenal Syndrome Type 1 (HRS-1)

Rhea-AI Summary

Mallinckrodt plc (NYSE: MNK) announced that the FDA's Cardiovascular and Renal Drugs Advisory Committee will hold a virtual meeting to review its New Drug Application (NDA) for terlipressin. This investigational drug targets hepatorenal syndrome type 1 (HRS-1), a severe condition with high mortality rates and no approved therapies in the U.S. The NDA is supported by data from the Phase 3 CONFIRM trial, the largest study of its kind for HRS-1, involving 300 patients. If approved, terlipressin could meet a critical medical need for approximately 30,000 to 40,000 annual patients in the U.S.

Positive

• FDA committee review indicates progress in terlipressin's NDA process.
• Terlipressin addresses a significant unmet medical need in HRS-1.
• NDA based on the largest prospective study (n=300) conducted for HRS-1.

Negative

• Terlipressin is still under investigation and not yet approved in the U.S. and Canada.

STAINES-UPON-THAMES, United Kingdom, July 7, 2020 /PRNewswire/ -- Mallinckrodt plc (NYSE: MNK), a global biopharmaceutical company, today announced that the Cardiovascular and Renal Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) will, as expected, hold a virtual meeting to review data on terlipressin, an investigational agent being evaluated for the treatment of hepatorenal syndrome type 1 (HRS-1). The company announced the FDA accepted for review its New Drug Application (NDA) for terlipressin in April. 

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 look forward to engaging the advisory committee panel in a robust discussion of the clinical evidence to support the safety and efficacy profile of terlipressin," said Steven Romano, M.D., Executive Vice President and Chief Scientific Officer at Mallinckrodt. "And we are extremely grateful to all the patients and healthcare professionals who made the data collection possible. If approved, we believe terlipressin has the potential to address a critical unmet need in HRS-1."

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

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.

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
This release includes forward-looking statements with regard to terlipressin, including the anticipated regulatory review process 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.

CONTACT

For Trade Media Inquiries
Caren Begun
Green Room Communications
201-396-8551
caren@greenroompr.com

For Financial/Dailies Media Inquiries
Ron Bartlett
H+K Strategies
Senior Vice President
813-545-2399
ron.bartlett@hkstrategies.com 

Investor Relations
Daniel J. Speciale, CPA
Vice President, Finance and IRO
314-654-3638
daniel.speciale@mnk.com

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-2000671 06/20

References

¹ National Organization for Rare Disorders. Hepatorenal Syndrome. Available at: -1%26h%3D1692784539%26u%3Dhttps%253A%252F%252Frarediseases.org%252Frare-diseases%252Fhepatorenal-syndrome%252F%26a%3Dhttps%253A%252F%252Frarediseases.org%252Frare-diseases%252Fhepatorenal-syndrome%252F&a=https%3A%2F%2Frarediseases.org%2Frare-diseases%2Fhepatorenal-syndrome%2F" rel="nofollow">https://rarediseases.org/rare-diseases/hepatorenal-syndrome/. Accessed June 12, 2020.
² 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. 
³ Gines P, Sola E, Angeli P, et al. Hepatorenal syndrome. Nature Reviews. (2018) 4:23. 
⁴ 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. 
⁵ 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.
⁶ United States Census Bureau: Quick Facts. Available at: https://www.census.gov/quickfacts/fact/table/US/PST045218. Accessed June 12, 2020.

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SOURCE Mallinckrodt Pharmaceuticals

FAQ

What is terlipressin and its significance for MNK shareholders?

Terlipressin is an investigational drug for the treatment of hepatorenal syndrome type 1 (HRS-1). Its approval could address a major medical need and positively impact MNK's stock performance.

When will the FDA Advisory Committee review terlipressin?

The FDA's Advisory Committee will hold a virtual meeting to review terlipressin, although the exact date has not been specified in the announcement.

What are the results of the Phase 3 CONFIRM trial for terlipressin?

The Phase 3 CONFIRM trial is the largest-ever study of HRS-1, involving 300 patients. Its results support the NDA for terlipressin.

How many patients with HRS-1 are there in the U.S.?

It is estimated that HRS-1 affects between 30,000 and 40,000 patients in the U.S. annually.

Why is the FDA review of terlipressin important for Mallinckrodt?

The FDA review is crucial as it could lead to the approval of terlipressin, potentially increasing revenue and enhancing shareholder value for Mallinckrodt.