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

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• Publié par: takeda
• Nombre de lectures: 14
• Langue: English

Extrait

Takeda Receives Positive CHMP Opinion for

Teduglutide (Revestive®) for Patients with

Short Bowel Syndrome

PR Newswire

OSAKA, Japan and BEDMINSTER, New Jersey, June 22, 2012

OSAKA, Japan

and

BEDMINSTER, New Jersey

,

June 22, 2012

/PRNewswire/ --

European CHMP adopts positive opinion for teduglutide (Revestive®) 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 (TSE:4502, "Takeda") and NPS

Pharmaceuticals, Inc. (NASDAQ: NPSP, "NPS"), 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

®

) 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

®

) 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

®

)

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. (NASDAQ: NPSP), 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

®

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® (teduglutide) as a treatment

for adult short bowel syndrome (SBS) and a Phase 3 registration study has been

completed for Natpara™ (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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