STAINES-UPON-THAMES, United
Kingdom, March 17, 2020
/PRNewswire/ -- Mallinckrodt
plc (NYSE: MNK), a global biopharmaceutical company, today
announced the completion of its rolling submission of a New Drug
Application (NDA) to the U.S. Food and Drug Administration (FDA)
for terlipressin, an investigational agent being evaluated for the
treatment of hepatorenal syndrome type 1 (HRS-1). The company
previously announced the rolling submission, which it initiated in
February.
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 less than 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
"Completion of the NDA submission for terlipressin is another
important step forward in our goal of bringing the first
FDA-approved therapeutic option to patients in the U.S. with HRS-1
– a life-threatening, difficult-to-treat condition2,"
said Steven Romano, M.D.,
Executive Vice President and Chief Scientific Officer
at Mallinckrodt. "We look forward to working with the FDA
during the regulatory review process in the coming months, and are
grateful to the patients, caregivers and investigators who
participated in our clinical trial."
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.
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
Jim Heins
H+K Strategies
212-885-0463
jim.heins@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-2000451 03/20
References
1 National Organization for Rare Disorders.
Hepatorenal Syndrome. Available
at: https://rarediseases.org/rare-diseases/hepatorenal-syndrome/.
Accessed April 9, 2019.
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 US Census 2018
https://www.census.gov/search-results.html?searchType=web&cssp=SERP&q=US
population 2018, Accessed on August 6,
2019.
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 March 13,
2020.
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SOURCE Mallinckrodt Pharmaceuticals