Market Overview

Shionogi Announces FDA Approval of Mulpleta® (Lusutrombopag)

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Decision Marks Latest Once-Daily, Oral Treatment of
Thrombocytopenia in Adults with Chronic Liver Disease Scheduled to
Undergo a Procedure

Shionogi & Co., Ltd. (hereafter "Shionogi") announced today that, after
completing a Priority Review, the U.S. Food and Drug Administration
(FDA) has approved Mulpleta® (lusutrombopag), a
once-daily, orally administered, small molecule thrombopoietin (TPO)
receptor agonist for the treatment of thrombocytopenia in adult patients
with chronic liver disease (CLD) who are scheduled to undergo a
procedure.

"We are very pleased the FDA has approved Mulpleta as a new, safe and
effective treatment," said Takayuki Yoshioka, President and Chief
Executive Officer, Shionogi Inc. "This new therapy offers physicians and
patients another choice beyond platelet transfusions as adult patients
with CLD often undergo procedures that could put them at increased risk
for bleeding."

The FDA approval was based on consistent safety and efficacy data from
two Phase 3 clinical trials, L-PLUS 1 and L-PLUS 2, in which Mulpleta
met primary and secondary endpoints with statistically significant
results.

"Therapeutic options in this area are critically needed, as adult
patients with chronic liver disease often require multiple procedures
for various medical reasons," said Dr. Nezam Afdhal, Senior Physician in
Hepatology at Beth Israel Deaconess Medical Center and Professor of
Medicine at Harvard Medical School. "I look forward to being able to
offer this new oral treatment to patients, instead of relying solely on
platelet transfusion."

About Thrombocytopenia in Chronic Liver Disease

Thrombocytopenia is a common complication of CLD, which may be caused by
multiple factors including decreased production of TPO. Thrombocytopenia
is frequently observed in patients with CLD, with studies suggesting
that it occurs in up to 78% of patients with cirrhosis.1 CLD-associated
thrombocytopenia is defined as a platelet count of less than 150,000/µL
and is the most common hematologic complication of CLD.2,3,4 Patients
with CLD and thrombocytopenia are at increased risk for bleeding,
requiring recurrent platelet transfusions, increased ambulatory visits
and inpatient hospital stays compared with patients with CLD without
thrombocytopenia.5 The annual health care cost of a patient
with CLD with thrombocytopenia is more than three times that of a
patient with CLD without thrombocytopenia.5 In addition to
the potential of thrombocytopenia, especially severe thrombocytopenia
(platelet count less than 50,000/µL), to aggravate surgical or traumatic
bleeding, it may also significantly complicate routine diagnostic
procedures and patient care, such as liver biopsy and medically
indicated or elective procedures for cirrhotic patients, resulting in
delayed or cancelled curative treatment.6

About Mulpleta

Mulpleta (lusutrombopag) is a once-daily, orally administered, small
molecule TPO receptor agonist for the treatment of thrombocytopenia in
adult patients with CLD who are scheduled to undergo a procedure.
Lusutrombopag is an orally bioavailable, small molecule TPO receptor
agonist that interacts with the transmembrane domain of human TPO
receptors expressed on megakaryocytes to induce the proliferation and
differentiation of megakaryocytic progenitor cells from hematopoietic
stem cells and megakaryocyte maturation.

Mulpleta was approved by the Ministry of Health, Labor and Welfare in
Japan in September 2015 for the improvement of thrombocytopenia
associated with CLD in patients undergoing an elective invasive
procedure. The European Medicines Agency has validated for review
Shionogi's standard Marketing Authorization Application for
lusutrombopag and approval is expected in 1H 2019.

In the U.S., Mulpleta is expected to be available by early September
2018.

Please see Important Safety Information, including Warnings &
Precautions and Adverse Reactions below
.

INDICATION

Mulpleta® (lusutrombopag) is indicated for the
treatment of thrombocytopenia in adult patients with chronic liver
disease who are scheduled to undergo a procedure.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Mulpleta is a thrombopoietin (TPO) receptor agonist, and TPO receptor
agonists have been associated with thrombotic and thromboembolic
complications in patients with chronic liver disease. Portal vein
thrombosis has been reported in patients with chronic liver disease
treated with TPO receptor agonists.

Consider the potential increased thrombotic risk when administering
Mulpleta to patients with known risk factors for thromboembolism.
Monitor platelet counts and for thromboembolic events and institute
treatment promptly.

Mulpleta should not be administered to patients with chronic liver
disease in an attempt to normalize platelet counts.

ADVERSE REACTIONS

The most common adverse reaction (≥ 3%) with Mulpleta was headache.

About Shionogi

Shionogi & Co., Ltd. is a major research-driven pharmaceutical company
dedicated to bringing benefits to patients based on its corporate
philosophy of "supplying the best possible medicine to protect the
health and wellbeing of the patients we serve." Shionogi's research and
development currently target two therapeutic areas: infectious diseases,
and pain/CNS disorders. For over 50 years, Shionogi has developed and
commercialized innovative oral and parenteral anti-infectives. In
addition, Shionogi is engaged in new research areas, such as
obesity/geriatric metabolic disease and oncology/immunology.
Contributing to the health and quality of life of patients around the
world through development in these therapeutic areas is Shionogi's
primary goal. For more details, please visit www.shionogi.co.jp/en/.
For more information on Shionogi Inc., the U.S.–based subsidiary of
Shionogi & Co., Ltd., headquartered in Florham Park, NJ, USA, please
visit www.shionogi.com.
For more information on Shionogi Ltd., the UK-based subsidiary of
Shionogi & Co. Ltd., headquartered in London, England, please visit www.shionogi.eu.

Forward Looking Statement

This announcement contains forward-looking statements. These
statements are based on expectations in light of the information
currently available, assumptions that are subject to risks and
uncertainties which could cause actual results to differ materially from
these statements. Risks and uncertainties include general domestic and
international economic conditions such as general industry and market
conditions, and changes of interest rate and currency exchange rate.
These risks and uncertainties particularly apply with respect to
product-related forward-looking statements. Product risks and
uncertainties include, but are not limited to, completion and
discontinuation of clinical trials; obtaining regulatory approvals;
claims and concerns about product safety and efficacy; technological
advances; adverse outcome of important litigation; domestic and foreign
healthcare reforms and changes of laws and regulations. Also for
existing products, there are manufacturing and marketing risks, which
include, but are not limited to, inability to build production capacity
to meet demand, unavailability of raw materials and entry of competitive
products. The company disclaims any intention or obligation to update or
revise any forward-looking statements whether as a result of new
information, future events or otherwise.

References:

     

1.

 

Peck-Radosavljevic M. Liver Int. 2017; 37(6):778-793.

2.

Giannini EG. Aliment Pharmacol Ther. 2006; 23(8):1055-1065.

3.

Koruk M, et al. Hepatogastroenterology. 2002; 49(48):1645-1648.

4.

Aref S, et al. Hematology. 2004; 9(5/6):351-356.

5.

Poordad F, et al. J Med Econ. 2012; 15:112-124.

6.

Hayashi H, et al. World J Gastroenterol. 2014; 20: 2595-2605.

USMUL-0003

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