Takeda Receives Positive CHMP Opinion for Teduglutide (Revestive®) for Patients with Short Bowel Syndrome

Osaka, Japan And Bedminster, New Jersey (ots/PRNewswire) -

- European CHMP adopts positive opinion for teduglutide (Revestive(R)) for
patients with short bowel syndrome (SBS)
- SBS is a rare and highly disabling condition that impacts patients' quality of
life and can lead to serious life-threatening complications
- Teduglutide has received orphan drug designation for the treatment of SBS from
the European Medicines Agency (EMA) and U.S. Food and Drug Administration (FDA)

Takeda Pharmaceutical Company Limited and NPS Pharmaceuticals,
Inc. , jointly announced today that the Committee for Medicinal
Products for Human Use (CHMP) of the European Medicines Agency has
adopted a positive opinion, recommending the granting of a marketing
authorisation for the medicinal product teduglutide (tradename in
Europe: Revestive(R)) as a once-daily treatment for adult patients
with short bowel syndrome (SBS). The marketing authorisation
application was submitted in March 2011.

SBS is a rare and debilitating disease characterised by the
body's severely impaired ability to absorb nutrients and fluids
through the gastrointestinal tract in people who have had a
significant portion of their small intestine removed. SBS typically
arises after extensive surgical resection of the bowel due to Crohn's
disease, ischemia or other conditions. Many patients with SBS depend
on chronic parenteral nutrition (PN) and/or intravenous (IV) fluids
to survive. There are currently no therapies approved for the
treatment of SBS in Europe.

Teduglutide (Revestive(R)) is a novel, recombinant analogue of
human glucagon-like peptide 2 (GLP-2), a naturally occurring protein
involved in the rehabilitation of the intestinal lining. Teduglutide
has received orphan drug designation for the treatment of SBS from
the European Medicines Agency (EMA) and U.S. Food and Drug
Administration (FDA).

"Short bowel syndrome patients suffer from malnutrition and
diarrhoea, and often parenteral nutrition is necessary to maintain
life," said Professor Palle Bekker Jeppesen, M.D., Ph.D, Department
of Medical Gastroenterology, Rigshospitalet, University Hospital of
Copenhagen, Denmark. "Revestive is a new, unique and important
treatment option for our patients and is adding important value to
the limited treatment armamentarium."

"We welcome the positive opinion from the CHMP for teduglutide.
This is good news for patients with SBS," said Trevor Smith, Head of
Commercial Operations, Europe & Canada, of Takeda.

"We are pleased with the Committee's recommendation, which brings
us closer to our goal of making teduglutide available in Europe for
patients with short bowel syndrome," said Francois Nader, MD,
President and Chief Executive Officer of NPS Pharmaceuticals.
"Teduglutide represents an important treatment advance that could
significantly reduce or even eliminate parenteral nutrition support
for patients with short bowel syndrome. We congratulate our partner
Takeda on receiving this positive opinion and look forward to
supporting their efforts to bring this much-needed therapy to
patients."

The CHMP opinion was based upon data from STEPS, the pivotal
phase-3 double-blind, placebo-controlled study in patients with SBS,
who required parenteral nutrition; 43-patients were randomised to a
subcutaneous 0.05-mg/kg/day dose of teduglutide and 43-patients to
placebo for up to 24-weeks.[1]

The proportion of teduglutide treated subjects achieving a 20% to
100% reduction of parenteral nutrition at Week-20 and 24 was
statistically significantly different from placebo, (63% versus 30%,
p=0.002). Treatment with teduglutide resulted in a 4.4-l/week
reduction in parenteral nutrition requirements versus 2.3-l/week for
placebo at 24-weeks (p<0.001). 21 patients treated with teduglutide
(54%) versus nine on placebo (23%) achieved at least a one day
reduction in parenteral nutrition administration (p=0.005)
Teduglutide was well-tolerated in the doses, frequency, and duration
of treatment used in this study.[1]

In another phase-3 double-blind, placebo-controlled study in
patients with SBS, who required parenteral nutrition, patients
subcutaneously received a 0.05-mg/kg/day dose (n-=-35), a
0.10-mg/kg/day dose (n-=-32) of teduglutide or placebo (n-=-16) for
up to 24-weeks.[2]

The primary efficacy analysis of the study results showed no
statistically significant difference between the group on teduglutide
0.10-mg/kg/day and the placebo group, while the proportion of
subjects receiving the recommended teduglutide dose of 0.05-mg/kg/day
achieving at least a 20% reduction of parenteral nutrition at Week-20
and-24 was statistically significantly different versus placebo (46%
versus 6%, p<0.01). Treatment with teduglutide resulted in a
2.5-l/week reduction in parenteral nutrition requirements versus
0.9-l/week for placebo at 24-weeks (p=0.08). Teduglutide was well
tolerated at the 0.05 mg/kg daily dose for the duration of treatment
of adult SBS subjects.[2]

About Short Bowel Syndrome

Short bowel syndrome (SBS) is a highly disabling condition that
can impair a patient's quality of life and lead to serious
life-threatening complications. SBS typically arises after extensive
resection of the bowel due to Crohn's disease, ischemia or other
conditions. SBS patients often suffer from malnutrition, severe
diarrhoea, dehydration, fatigue, osteopenia, and weight loss due to
the reduced intestinal capacity to absorb nutrients, water, and
electrolytes. The usual treatment for short bowel syndrome is
nutritional support, including parenteral nutrition (PN) and/or
intravenous (IV) fluids to supplement and stabilize nutritional
needs.

Although PN can provide nutritional support for short bowel
syndrome patients, it does not improve the body's own ability to
absorb nutrients. PN is also associated with serious complications,
such as infections, blood clots or liver damage, and the risks
increase the longer patients are on PN. Patients on PN often
experience a poor quality of life with difficulty sleeping, frequent
urination and loss of independence.

About Teduglutide (Revestive(R))

Teduglutide is a novel, recombinant analogue of human
glucagon-like peptide 2, a protein involved in the rehabilitation of
the intestinal lining. It has been developed to reduce dependence on
parenteral nutrition (PN) in adult patients with short bowel syndrome
(SBS). Two phase III studies of teduglutide demonstrated a favorable
safety profile and significant reductions in mean PN volume from
baseline to end of treatment. In addition some patients were able to
be weaned off PN and continue their life without parenteral support

Teduglutide has received orphan drug designation for the
treatment of SBS from the European Medicines Agency (EMA) and U.S.
Food and Drug Administration (FDA).

In 2007, NPS Pharmaceuticals, Inc. , a specialty pharmaceutical
company developing innovative therapeutics for rare gastrointestinal
and endocrine disorders, granted Nycomed the rights to develop and
commercialize teduglutide outside the United States, Canada and
Mexico and Israel. NPS retains all rights to teduglutide in North
America. NPS submitted a new drug application for teduglutide to the
U.S. Food and Drug Administration (FDA) in November 2011. Teduglutide
is known as Gattex(R) in the U.S.

References

1) Publication of STEPS is in preparation. First results were report at the
AGA 2011 in an abstract: Jeppesen PB, Pertkiewicz M, Seidner DL, O'Keefe S, Heinze H,
Joelsson B: Teduglutide, a novel analogue of Glucagon-like Peptide 2 (GLP-2), is
effective and safe in reducing parenteral support volume in short bowel
syndrome-intestinal failure subjects: Results from a 24-week, placebo-controlled phase
3 trial (STEPS), Gastroenterology 2011; 140 (5), Supplement 1, S146
2) Jeppesen PB, Gilroy R, Pertkiewicz M, Allard JP, Messing B, O'Keefe SJ.
Randomised placebo-controlled trial of teduglutide in reducing parenteral nutrition
and/or intravenous fluid requirements in patients with short bowel syndrome. Gut. 2011
Jul;60(7):902-14. Epub 2011 Feb 11.

About Takeda Pharmaceuticals Company Limited

Located in Osaka, Japan, Takeda is a research-based global
company with its main focus on pharmaceuticals. As the largest
pharmaceutical company in Japan and one of the global leaders of the
industry, Takeda is committed to strive towards better health for
patients worldwide through leading innovation in medicine. Additional
information about Takeda is available through its corporate website,
http://www.takeda.com.

Additional information about Takeda is available through its
corporate website, http://www.takeda.com.

About NPS Pharmaceuticals

NPS Pharmaceuticals is a biopharmaceutical company focused on
bringing orphan products to patients with rare disorders and few, if
any, therapeutic options. NPS is advancing two late-stage
registration programs. A New Drug Application is undergoing FDA
review for Gattex(R) (teduglutide) as a treatment for adult short
bowel syndrome (SBS) and a Phase 3 registration study has been
completed for Natpara(TM) (recombinant human parathyroid hormone
(rhPTH [1-84]) in adult hypoparathyroidism. NPS' earlier stage
pipeline includes two calcilytic compounds, NPSP790 and NPSP795, with
potential application in rare disorders involving increased calcium
receptor activity, such as autosomal dominant hypocalcemia with
hypercalciuria (ADHH). NPS complements its proprietary programs with
a royalty-based portfolio of products and product candidates that
includes agreements with Amgen, GlaxoSmithKline, Janssen
Pharmaceuticals, Kyowa Hakko Kirin, and Nycomed (acquired by Takeda
Pharmaceutical Company Limited).

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ots Originaltext: Takeda Chemical Industries
Im Internet recherchierbar: http://www.presseportal.de

Contact:
Media Contacts, Takeda Pharmaceutical Company Limited,

Public Relations and Investor Relations, +81-3-3278-2037;

European media, Tobias Cottmann, +41445551510,
tobias.cottmann@takeda.com;
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