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
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
M: +1 813 545 2399
ron.bartlett@hkstrategies.com
Investor Relations
Daniel J.
Speciale, CPA
Vice President, Investor Relations 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-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.
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. J Investig
Med 2016;64:33–38.
6 United States Census Bureau: Quick Facts.
Available at:
https://www.census.gov/quickfacts/fact/table/US/PST045218. Accessed
April 20, 2020.
7 U.S. Food and Drug Administration. "Fast Track".
Available at
https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/fast-track.
Accessed April 20, 2020.
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SOURCE Mallinckrodt Pharmaceuticals