Gattex® (teduglutide) is a drug for the treatment of short bowel syndrome (SBS). Short bowel syndrome is a rare condition related to poor absorption of nutrients. It typically occurs in people who have had half or more of their small intestine removed, and cannot absorb enough water, vitamins, and other nutrients from food. They may then need to use parenteral nutrition (PN) and intravenous (IV) fluids, the slow infusion of a solution of nutrients and fluids into a vein.
Gattex works by regeneration of cells in the intestinal lining, slowing down transit through the gut and increasing blood flow, allowing for increased nutrient absorption. In studies, the drug was associated with achieving and maintaining clinically meaningful reductions in PN and IV fluid volume in adult subjects with short bowel syndrome.
Participants Sought for a Short Bowel Syndrome (SBS) Research Study for Children Up To 17 Years of Age on Parenteral Nutrition
Description: Short bowel syndrome is a rare disorder. This 24-week, double-blind study is evaluating the safety and effectiveness of teduglutide in children up to the ages of 17 with short bowel syndrome (SBS). Teduglutide is currently an approved medicine for adults with SBS. The aim of the study is to increase absorption of nutrients which may result in decreased parenteral support.
Study Population: The study is in a pediatric patient population of males and females with SBS dependent on Parenteral Support – the upper age limit is 17 years of age. The study will take place in the US, Canada, Belgium, Finland, Italy, UK, France, and Germany.
Phase: Phase III
Participants Sought for a Long-term Study of Patients with Short Bowel Syndrome (SBS)
March 3, 2014 – This global clinical study has begun enrolling patients with SBS in order to provide additional long-term data on safety of teduglutide and on the natural history of SBS in patients in routine, real world settings. The information gathered is intended to assist health care providers in optimizing their clinical decision-making in managing SBS patients.
January 5, 2015 – The Japanese Ministry of Health, Labor, and Welfare (MHLW) granted teduglutide (Gattex in the US; Revestive in the EU) orphan drug status for the treatment of adult patients with short bowel syndrome (SBS).
June 30, 2014 – The U.S. Food and Drug Administration (FDA) has approved updated labeling for Gattex (teduglutide [rDNA origin]) for injection, to include long-term data from adult patients with Short Bowel Syndrome (SBS). The data, published in 2013, demonstrated that there was an increased response to treatment over time in all groups receiving Gattex. The revised labeling provides this important information for healthcare professionals and patients about long-term use of Gattex.
Supplemental New Drug Application Accepted for Gattex (teduglutide)
November 13, 2013 — The FDA has accepted for consideration a supplemental New Drug Application (sNDA) for Gattex. The application seeks to include new, long term usage data on the product label. This new data, from a 2 year study presented at the ACG meeting in October, showed that patients using Gattex beyond one year continued to be able to reduce their support on parenteral nutrition.
October 24, 2013 — New findings from a two-year study by NPS Pharmaceuticals support the long-term use of Gattex (teduglutide [rDNA origin]) for injection in adult patients with Short Bowel Syndrome (SBS). The data were presented at the American College of Gastroenterology (ACG) Annual Scientific Meeting and Postgraduate Course in San Diego, CA in October 2013.
The open-label extension study included 88 adult patients with SBS. Investigators reported that the long-term use of Gattex in patients with SBS resulted in additional, clinically meaningful reductions in the volume and days per week of parenteral support requirements in this extension study. Thirteen (13) patients in the study achieved complete independence from parenteral support with long-term Gattex therapy. No new unexpected safety concerns were observed with long-term Gattex treatment and the product’s safety profile remains consistent with the product’s label.
This study was published as an abstract and presented at a conference. These data and conclusions should be considered to be preliminary until published in a peer-reviewed journal. The poster, entitled “Long-term Safety and Efficacy of Teduglutide for the Treatment of Intestinal Failure Associated with Short Bowel Syndrome: Final Results of the Steps-2 Study, a 2-year, Multicenter, Open-label Clinical Trial,” was recognized as an ACG Presidential Poster Award recipient, which identifies the most highly-ranked abstracts in poster sessions.
Study Reports Safety and Efficacy of Teduglutide over 52 Week Treatment Period
January 24, 2013 – An international multi-center study involving 52 patients looked at the safety, tolerability, and efficacy of teduglutide (Gattex) taken once per day over 52 weeks for the treatment of people with short bowel syndrome receiving parenteral nutrition (PN). The study concluded that for patients with short bowel syndrome and intestinal failure, the efficacy of teduglutide was maintained over 52 weeks and the safety profile was sufficient for it to be considered for long-term use.
A reduction of more than 20%, equivalent to 1 day per week, of parenteral nutrition dependency was achieved in a significant number of participants receiving the drug. Four (4) patients were able to discontinue PN fluids completely over the 52 weeks.
The most common adverse events reported included headache, nausea, and abdominal pain. Of 7 patients who withdrew because of adverse events, 4 were considered treatment related. While the researchers found the safety profile to be acceptable, they advise that physicians closely monitor patients beginning the drug for side effects and possible need to adjust dose.
Patients taking part in clinical trials are closely monitored. Continued clinical practice studies are needed to understand real world implications for improving quality of life and reducing potential long-term PN complications for people with short bowel syndrome.
The study titled, “Safety and efficacy of teduglutide after 52 weeks of treatment in patients with short bowel syndrome intestinal failure,” was reported in the journal, Clinical Gastroenterology and Hepatology, Jan 17, 2013.
FDA Approves Gattex to Treat Short Bowel Syndrome
December 21, 2012 – The U.S. Food and Drug Administration (FDA) today approved Gattex (teduglutide) for the treatment of adult patients with short bowel syndrome (SBS) who are dependent on parenteral support. Gattex will be available in the first quarter of 2013. To help ensure that the benefits of Gattex outweigh the risks for causing other serious conditions, the drug is approved with a Risk Evaluation and Mitigation Strategy, which patients need to discuss with their doctors.
The New Drug Application for Gattex to Treat Short Bowel Syndrome is under Review by the U.S. Food and Drug Administration (FDA)
On October 16, 2012, the FDA’s Gastrointestinal Drugs Advisory Committee voted unanimously to recommend approval of Gattex for adults with SBS. The drug is currently under review by the FDA, which is expected to make a final decision on the drug by this year end.
Gattex is a novel, recombinant analog of human glucagon-like peptide 2, a peptide involved in gastrointestinal regeneration and repair. NPS Pharmaceuticals is a specialty pharmaceutical company developing orphan therapeutics for rare gastrointestinal and endocrine disorders.
The company's SBS clinical development program represents the largest and most comprehensive to date. The information in the NDA is derived from 14 completed and one ongoing clinical study. A total of 566 subjects have been treated with teduglutide. Of the 566 Gattex-treated subjects, 97 SBS subjects had at least 12 months of exposure to Gattex. Across the company’s Phase 3 studies, a total of 15 patients were able to achieve independence from PH/IV. Gattex may potentially cause other serious condtions. The most common side effects include abdominal pain, upper respiratory tract infections, nausea, injection site reactions, headaches, gastrointestinal stoma complications, and abdominal distension.
SBS is a rare disorder characterized by inadequate absorption of fluids and nutrients in people who have had a significant portion of their small intestine surgically removed due to disease or injury. It is a highly disabling condition that can lead to serious life-threatening complications and impair a patient's quality-of-life. SBS typically arises after extensive resection of the bowel due to Crohn's disease, ischemia, or other conditions. The condition is associated with intestinal failure and the inability to absorb sufficient nutrients and/or fluids through the gastrointestinal tract.
People with SBS are highly prone to malnutrition, diarrhea, dehydration, and an inability to maintain weight due to the reduced intestinal capacity to absorb macronutrients, water, and electrolytes. As a result, many patients require the long-term use of parenteral nutrition (PN) and intravenous (IV) fluids to supplement their nutritional needs and stabilize their hydration. Although PN/IV can meet basic nutrition and fluid requirements, it does not improve the body's ability to absorb nutrients.
The long-term use of PN/IV fluids is associated with serious and life-threatening complications. Patients on parenteral support often experience a poor quality of life with difficulty sleeping, frequent urination, and loss of independence.
Learn more about short bowel syndrome here.
What are Phases?
Treatment trials or studies are in phases:
- Phase 1 tests a new drug or treatment in a small group to evaluate its safety, determine a safe dosage range, and identify side effects;
- Phase 2 expands the study to a larger group of people to see if it is effective and to further evaluate its safety;
- Phase 3 expands the study to an even larger group of people to confirm its effectiveness, monitor side effects, and collect information that will allow the drug or treatment to be used safely.