DUBLIN, July 15, 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) voted to recommend approval for its
investigational agent terlipressin to treat adults with hepatorenal
syndrome type 1 (HRS-1) (8 yes; 7 no). HRS-1 is an acute and
life-threatening syndrome involving acute kidney failure in people
with cirrhosis.1 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.
"Mallinckrodt is pleased with the
advisory committee's positive vote in favor of approval for
terlipressin, supporting the potential clinical value terlipressin
can bring to patients with HRS-1 in need of an approved therapy in
the U.S.," said Steven Romano,
M.D., Executive Vice President and Chief Scientific Officer
at Mallinckrodt. "We acknowledge the clinical challenges
associated with treating this complex disease in such a critically
ill patient population. We are committed to working closely with
the FDA as it continues to review our application."
HRS-1 requires a diagnosis of exclusion, and is often a
challenge to diagnose in a timely manner.2 If left
untreated, HRS-1 has a median survival time of approximately two
weeks and greater than 80 percent mortality within three
months.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 Americans
annually.5,6
"Terlipressin plus albumin is the recommended standard-of-care
therapy for HRS-1 in many other countries, where terlipressin is
approved," said Francois Durand,
M.D., on behalf of the International Club of Ascites. "HRS-1 is
one of the most severe complications of end stage liver disease and
the favorable vote from the advisory committee is encouraging to
U.S. patients in need of treatment."
While recommendations of the advisory committee are not binding,
the FDA will consider the advice provided by the committee as part
of the New Drug Application (NDA) review. The FDA assigned
terlipressin a Prescription Drug User Fee Act (PDUFA) target date
of September 12, 2020. The company announced the FDA accepted
for review its NDA for terlipressin in April
2020, which was 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 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 regulatory review
process and related timing, as well as 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 Media Inquiries
Caren
Begun
Green Room Communications
201-396-8551
caren@greenroompr.com
Investor Relations
Daniel J.
Speciale, CPA
Vice President, Finance and Investor Relations Officer
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-2001138 07/20
References
1 National Organization for Rare Disorders.
Hepatorenal Syndrome. Available
at: https://rarediseases.org/rare-diseases/hepatorenal-syndrome/.
Accessed June 3, 2020.
2 Gines P, Sola E, Angeli P, et al. Hepatorenal
syndrome. Nature Reviews. (2018) 4:23.
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 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 3, 2020.
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