STAINES-UPON-THAMES, United
Kingdom, March 2, 2020
/PRNewswire/ -- Mallinckrodt
plc (NYSE: MNK), a global biopharmaceutical company, today
announced it initiated the rolling submission of a New Drug
Application (NDA) to the U.S. Food and Drug Administration (FDA)
for terlipressin, an investigational agent being investigated for
the treatment of hepatorenal syndrome type 1 (HRS-1), by submitting
its clinical data package. A rolling submission allows the
company to submit portions of the regulatory application to the FDA
as they are completed.1 The company expects to complete
the submission of the NDA in the coming months.
HRS-1 is an acute and life-threatening syndrome involving acute
kidney failure in people with cirrhosis.2 The condition
has a median survival time of less than two weeks and greater than
80 percent mortality within three months if left
untreated.3,4 At present, there are no approved drug
therapies for HRS-1 in the U.S.,5 and it is estimated to
affect between 30,000 and 40,000 patients in the
U.S. annually.6,7
"This is an important milestone that brings us closer to
potentially delivering the first FDA-approved treatment in the U.S.
for HRS-1, a life-threatening, difficult-to-treat condition,"
2 said Steven Romano,
M.D., Executive Vice President and Chief Scientific Officer
at Mallinckrodt. "We are grateful to the patients, their
families and caregivers, and the researchers who made this
achievement possible."
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. The
CONFIRM trial met its primary endpoint of Verified HRS Reversal
(VHRSR), defined as renal function improvement, avoidance of
dialysis and short-term survival (p=0.012). Serious adverse events
were reported in 65 percent (n=130) of subjects in the terlipressin
group and 60.6 percent (n=60) in the placebo group. No new or
unexpected AEs were reported. 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 expected timing for
completion of Mallinckrodt's submission
to the FDA 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-2000024 02/20
References
1 U.S. Food and Drug Administration. "Guidance for
Industry Expedited Programs for Serious Conditions - Drugs and
Biologics." Available at
https://www.fda.gov/files/drugs/published/Expedited-Programs-for-Serious-Conditions-Drugs-and-Biologics.pdf.
Accessed February 6, 2020.
2 National Organization for Rare Disorders. Hepatorenal
Syndrome. Available
at: https://rarediseases.org/rare-diseases/hepatorenal-syndrome/.
Accessed April 9, 2019.
3 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.
4 Gines P, Sola E, Angeli P, et al. Hepatorenal
syndrome. Nature Reviews. (2018) 4:23.
5 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.
6 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.
7 US Census 2018
https://www.census.gov/search-results.html?searchType=web&cssp=SERP&q=US
population 2018, accessed on 06 August
2019.
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SOURCE Mallinckrodt Pharmaceuticals