Short Bowel Syndrome - Teduglutide Gets Positive Response, Europe

Date: Jun-25-2012Short Bowel Syndrome (SBS) is a highly debilitating condition, which occurs when the body's gastrointestinal tract is unable to absorb sufficient nutrients and fluids due to a resection of a significant portion of the bowel as a result of Crohn's disease, ischemia or other conditions.

Because the patient's body is unable to absorb insufficient nutrients, water and electrolytes, SBS patients often suffer from malnutrition, severe diarrhea, dehydration, fatigue, osteopenia, and weight loss.

The standard treatment for SBS consists of nutritional support, such as parenteral nutrition (PN) and/or intravenous (IV) fluids to supplement and stabilize nutritional needs. PN is linked to serious complications, including infections, blood clots or liver damage. These risks increase the longer patients are on PN. Patients on PN often experience a poor quality of life with frequent urination, difficulty sleeping and loss of independence. Until now, there have been no approved therapies available in Europe to treat SBS.

The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has now recommended that Takeda Pharmaceutical Company Limited and NPS Pharmaceuticals, Inc. are granted marketing authorization for teduglutide, which is known under the European trade name of Revestive®, as a once-daily treatment for adults with short bowel syndrome (SBS). The companies submitted their application for marketing authorization in March 2011 and the EMA and US FDA has now awarded Teduglutide with an orphan drug designation for the treatment of SBS.

Teduglutide (Revestive®) contains a novel, recombinant analogue of human glucagon-like peptide 2 (GLP-2), i.e. a natural protein that plays a role in rehabilitating the intestinal lining and has been designed to reduce dependence on parenteral nutrition (PN) in adults with SBS. The drug has been tested in two phase III studies, with both showing a favorable safety profile and substantial reductions in the average PN volume from the start of the studies until end of treatment. Furthermore, some patients could be weaned off PN and continue a life without parenteral support.

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

Trevor Smith, Head of Takeda's European and Canadian Commercial Operations said: "We welcome the positive opinion from the CHMP for teduglutide. This is good news for patients with SBS."

Francois Nader, MD, President and Chief Executive Officer of NPS Pharmaceuticals added:

"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. 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 based their decision to recommend teduglutide on two trials. The first pivotal phase 3 double-blind, placebo-controlled study involved a total of 86 SBS patients in need of parenteral nutrition, of which 43 patients were randomized to a subcutaneous 0.05 mg/kg/day dose of teduglutide whilst the remaining 43 patients were given placebo for up to 24 weeks.

The findings revealed that those in the teduglutide group achieved reductions between 20% to 100% of parenteral nutrition at Week 20 and 24, which was statistically substantially different compared with those in the placebo group, i.e. 63% compared with 30%, p=0.002. At 24 weeks, patients in the teduglutide group had a 4.4 l/week reduction in parenteral nutrition requirements compared with 2.3 l/week for placebo (pNot to be reproduced without permission of Medical News Today
Courtesy: Medical News Today Note: Any medical information available in this news section is not intended as a substitute for informed medical advice and you should not take any action before consulting with a health care professional.