Peptide education
Teduglutide
Gattex · ALX-0600
Teduglutide is a medicine that acts like GLP-2, a hormone involved in how the intestine adapts and absorbs fluid. The U.S. product, Gattex, is approved for people aged one year and older with short bowel syndrome who need nutrition or fluids through a vein, known as parenteral support.
Gattex can reduce the amount of intravenous support some patients need, but it does not guarantee independence from that support. Its use comes with monitoring for intestinal, fluid, biliary, pancreatic, and growth-related risks.
Overview
Quick answer
A need for parenteral support is part of the approved indication. A person with ordinary digestive symptoms or a desire to improve gut health is not the same population as the people studied for this drug.
What is it used for?
Gattex is approved for short bowel syndrome in adults and children aged one year and older who are dependent on parenteral support. The support may include intravenous fluids or nutrition.
What is the labeled dose?
The U.S. label specifies 0.05 mg per kilogram of body weight by subcutaneous injection once daily. For an estimated filtration rate below 60 mL/min/1.73 m2, it specifies 0.025 mg/kg once daily. The 5 mg kit is not recommended for pediatric patients weighing less than 10 kg.
Can it eliminate the need for intravenous support?
Some people in a longer open-label extension reached that outcome, while others continued to need support. The controlled trial measured reductions in support volume over 24 weeks.
What monitoring matters?
The label calls for age-specific gastrointestinal surveillance, periodic laboratory testing, and attention to fluid balance, obstruction, and other adverse effects. Absorption of some oral medicines may increase.
Evidence
Evidence snapshot
Gattex is approved for adults and children aged one year and older who meet the label's short bowel syndrome and support-dependence criteria.
In STEPS, 27 of 43 participants assigned to teduglutide and 13 of 43 assigned to placebo had at least a 20% reduction in support volume at both weeks 20 and 24. These are the randomized-group denominators.
Some participants achieved enteral autonomy. This extension had no concurrent placebo group, and response rates among completers exclude people who did not finish.
The label warns about possible acceleration of neoplastic growth, obstruction, fluid overload, biliary and pancreatic disease, and altered absorption of oral drugs.
Claims
Common claims vs evidence
| Claim | Human evidence | Mechanistic evidence | Verdict |
|---|---|---|---|
| Teduglutide can reduce parenteral support in short bowel syndrome. | The adult STEPS trial found more participants met its prespecified support reduction threshold with teduglutide than with placebo. | GLP-2 receptor activity affects intestinal adaptation and absorption. | Supported in a specific, support-dependent population. It does not mean every patient can stop intravenous nutrition or fluids. |
| Everyone who responds can stop parenteral support. | Some people reached enteral autonomy in an open-label extension, while others remained on support. The extension followed selected participants and did not include a concurrent placebo arm. | Not established. A reduction in support is different from complete independence from it. | |
| The approved drug is a general gut-repair or wellness treatment. | The approved indication and controlled trial concern short bowel syndrome with parenteral support dependence, not general digestive wellness. | The cited evidence does not support that broader claim. |
Bottom line
Main takeaway
In the adult STEPS trial, some people with short bowel syndrome reduced their need for intravenous fluids or nutrition.
Body weight, kidney function, age-specific surveillance, fluid balance, and medicines taken by mouth matter to the approved treatment context.
STEPS provides a placebo comparison over 24 weeks. STEPS-2 adds longer observation, but its open-label design and discontinuations limit inference.
Identity
What it is
Short bowel syndrome can leave too little functioning intestine to absorb adequate fluids or nutrients. Parenteral support supplies those needs through a vein; the amount required differs between patients.
Teduglutide is a 33-amino-acid GLP-2 analog. The approved U.S. product is a powder prepared for subcutaneous injection, and its label applies to specific patients who depend on parenteral support.
Use context
Routes, doses, and cycle patterns
Gattex uses weight-based dosing. The adult STEPS trial tested a daily subcutaneous dose over 24 weeks.
Human studies and product labels
Gattex U.S. label
- Purpose
- Short bowel syndrome with parenteral support dependence
- Context
- Approved product label for adults and children aged one year and older
- Route
- Subcutaneous injection
- Amount
- 0.05 mg/kg; 0.025 mg/kg when eGFR is below 60 mL/min/1.73 m2
- Frequency
- Once daily
- Duration
- Determined by clinical care
The label specifies a lower dose for patients below the stated kidney function threshold. Its 5 mg kit is not recommended for pediatric patients weighing less than 10 kg.
STEPS adult trial
- Purpose
- Reduction in parenteral support
- Context
- Randomized placebo-controlled study
- Route
- Subcutaneous injection
- Amount
- 0.05 mg/kg
- Frequency
- Once daily
- Duration
- 24 weeks
Investigators adjusted parenteral support using study criteria.
Research
Research evidence
The controlled adult STEPS trial measured support reductions over 24 weeks. A subsequent open-label extension followed participants for longer and found some reached enteral autonomy. The extension had no concurrent placebo group, and 65 of the 88 enrolled participants completed it.
| Study / evidence area | Population | Design | Product context | Main outcome | Limitations | Weight |
|---|---|---|---|---|---|---|
| STEPS | 86 adults with short bowel syndrome dependent on support at least three times weekly for at least 12 months | Randomized, double-blind, placebo-controlled trial | Daily subcutaneous teduglutide compared with placebo | At least a 20% reduction in weekly parenteral support at weeks 20 and 24 occurred in 27 of 43 participants assigned to teduglutide and 13 of 43 assigned to placebo, using the randomized-group denominators. | The trial lasted 24 weeks and involved a defined support-dependent population. | Controlled human trial |
| STEPS-2 | 88 adults enrolled in an extension of the STEPS program | Open-label extension | Subcutaneous teduglutide following screening or the original study | Sixty-five participants completed the extension; 13 participants achieved enteral autonomy across its groups. | There was no concurrent placebo group. Completion and previous treatment varied, so completer rates do not represent all who began the extension. | Longer-term observational trial follow-up |
Use context
Reported use context
In the STEPS trial, adults dependent on parenteral support were assigned to daily subcutaneous teduglutide or placebo for 24 weeks.
Study and label context
0.05 mg/kg once daily for 24 weeks, compared with placebo.
Cautions
Safety and unknowns
- The label warns that teduglutide may accelerate neoplastic growth. It describes gastrointestinal polyps and calls for surveillance. Treatment should be discontinued if active gastrointestinal malignancy develops.
- Adults are screened with colonoscopy and upper gastrointestinal endoscopy within six months before treatment. Follow-up colonoscopy and either upper gastrointestinal endoscopy or alternate imaging are recommended after one year and at least every five years thereafter, or more often when needed.
- Pediatric instructions differ: the label calls for stool blood testing before treatment and annually, with additional endoscopy when indicated, and recommends colonoscopy or sigmoidoscopy after one year and every five years during continuous treatment.
- Intestinal or stomal obstruction, gallbladder and pancreatic disease, fluid overload, and increased absorption of oral medicines are label concerns. Fluid and electrolyte imbalance can also occur after treatment stops.
Product quality
Product identity and quality
The evidence and instructions describe the approved Gattex formulation. A different vial or supplier is not established as equivalent by sharing the ingredient name.
Mechanism
How it is proposed to work
Teduglutide activates a gut-hormone pathway that can help the remaining intestine absorb more fluid. Effects on tissue growth also help explain why surveillance is part of the label.
The label places GLP-2 receptors in intestinal enteroendocrine cells, subepithelial myofibroblasts, and enteric neurons, with local mediator release following receptor activation.
A small dose-ranging study described in the label measured increased fluid absorption and changes in villus height and crypt depth at most studied doses.
FAQ
Common questions
Who is Gattex approved for in the United States?
Adults and children aged one year and older with short bowel syndrome who depend on parenteral support.
Does treatment mean parenteral support can always stop?
No. The randomized trial measured reductions in support, and only some patients in the open-label extension achieved independence from it.
Details
Technical details
Sources
References
- 1.
FDA. Gattex prescribing information
U.S. product indication, dosing, monitoring, and adverse reactions.
- 2.
doi:10.1053/j.gastro.2012.09.007 PMID:22982184
Randomized, placebo-controlled adult STEPS trial.
- 3.
doi:10.1038/ctg.2015.69 PMID:26844839
Open-label extension with completer and discontinuation limitations.