Short bowel syndrome (SBS) is a rare condition in which the body cannot absorb enough nutrients because part of the small intestine is missing or not working as well as it should be. This can lead to symptoms such as ongoing diarrhea, dehydration, and trouble maintaining proper nutrition. With treatment and time, some people with SBS get better. People’s remaining intestine changes, so they are able to absorb all the nutrients they need from regular food. But many people with SBS need medicine support while their intestines adapt and regain function.
Gattex (teduglutide) is a medicine that helps your intestines work better by improving its structure and nutrient absorption. This may help reduce the amount of intravenous (IV) fluid and IV nutrition (parenteral support) you need.
How Does Gattex Work for SBS?
Gattex is a medicine that acts like a natural hormone in your intestines, called glucagon-like peptide-2 (GLP‑2). The GLP-2 hormone plays a key role in helping your intestines absorb nutrients. GLP-2 helps the tiny finger‑like structures in the intestines, also known as villi, grow taller and deeper, which increases the surface area your body uses to absorb nutrients. GLP-2 also slows down the movement of food through your intestines, allowing more time for nutrients to be absorbed. Finally, GLP-2 improves the absorption of nutrients by reducing stomach acid and increasing blood flow to the intestines.
Gattex works by binding to GLP‑2 receptors in the intestines, leading to improved absorption of fluids and nutrients. Because of this improved absorption, if you use Gattex, you may be able to reduce the amount of parenteral support you need over time.
How Do I Use Gattex?
Gattex is a medicine that you inject under your skin once a day, usually at the same time every day. For children, Gattex must be given by a health care provider or a trained caregiver. It comes as a kit with a vial of Gattex powder, a syringe filled with sterile water, needles, and alcohol swabs. Before using anything in the kit, check the “Use By” date to make sure it has not passed. Do not use any part of the kit after its “Use By” date. You will likely be trained to use Gattex before you need to use the medicine at home. Refer to the product’s instructions for use that come with your medicine for detailed instructions with pictures. These instructions can also be found on the drugmaker’s website. If you have any questions about how to use Gattex, contact your health care provider.
The powder must be mixed with the sterile water before using it. After adding the sterile water to the vial with Gattex powder, let the vial sit for about 30 seconds, then gently roll it between your palms for about 15 seconds. Do not shake the vial. Let the mixed medicine stand for about two minutes. Check the vial for any powder that has not dissolved. If you see any leftover powder, gently roll it again until fully dissolved. Once mixed, the medicine must be injected within three hours. You should throw away any leftover medicine after you use your dose. Each vial can only be used once.
Gattex can be injected in the stomach, thigh, or upper arm. It is important to rotate the injection sites every day to avoid skin irritation.
How Was It Studied for SBS?
Gattex was tested in several clinical studies that included adults and children with SBS who needed IV nutrition or fluids.
Adults
The CL0600‑004 and the STEPS studies tested 24 weeks of daily Gattex in adults with SBS. In both studies, people who were enrolled relied on IV nutrition or fluids at least three times a week for at least a year before joining. Both studies included people from multiple countries. In the CL0600‑004 study, 83 participants were assigned to one of two doses of Gattex (0.05 or 0.10 mg per kg of body weight per day [mg/kg/day]) or a placebo (which has no medicine in it). In the STEPS study, 86 participants were assigned to Gattex 0.05 mg/kg/day or a placebo. In both studies, neither the participants nor the investigators knew which treatment each person was receiving.
The primary goal of both studies was to determine how many people could reduce the amount of IV nutrition or fluids (parenteral support volume) they needed by at least 20%.
At the start of the CL0600-004 study, the average age of the participants was 49 years, and the average length of time they had depended on parenteral nutrition was seven years (range, 1-24 years). The most common causes for intestinal surgery leading to SBS were Crohn’s disease (36%) and vascular disease (30%). In addition, 24 people (29%) had a stoma at the time they entered the study.
At the start of the STEPS study, the average age of the participants was 50 years, and the average length of time they had depended on parenteral nutrition was six years (range, 1-25 years). The most common causes for intestinal surgery leading to SBS were vascular disease (34%), Crohn’s disease (21%), and other causes (22%). In addition, 38 people (44%) had a stoma at the time they entered the study.
After completing the CL0600‑004 and STEPS studies, participants were allowed to continue in follow‑up extension studies.
The CL0600‑005 study was a 28‑week extension of the CL0600‑004 study. A total of 65 participants who had already used Gattex continued on the same dose in the CL0600‑005 study. People who had used a placebo were randomly assigned to start Gattex at a dose of 0.05 or 0.10 mg/kg/day.
STEPS‑2 was an extension study that followed the STEPS study. In STEPS‑2, 88 participants used Gattex at a dose of 0.05 mg/kg/day. People who had already used Gattex were treated with Gattex for a total of 30 months. Those who had used a placebo used Gattex for 24 months. After this, STEPS‑3 evaluated people for one more year of using Gattex.
Children
In children with SBS, Gattex was studied in a 24-week clinical study taking place in multiple countries. Enrolled children were of 1-17 years of age who needed ongoing IV nutrition or fluids. Before starting treatment, families chose whether the enrolled child would receive Gattex or continue standard care. Those who chose Gattex were randomly assigned to one of two daily doses (0.025 mg/kg or 0.05 mg/kg). Neither the families nor the investigators knew which Gattex dose was used.
The primary goal of the study was to determine how many children could reduce the amount of IV nutrition or fluids (parenteral support volume) they needed by at least 20%.
A total of 59 children enrolled. Fifty children got Gattex, and nine children continued standard care. Among children using Gattex, 24 got the 0.025 mg/kg dose and 26 got the 0.05 mg/kg dose. The average age of the children was between 6 and 7 years old. Most of the children were boys (69%). Race distribution varied and included White (66%), Black or African American (12%), Asian (5%), and other (5%); the race of some children was not reported (12%). The most common causes of SBS were gastroschisis (37%), midgut volvulus (32%), and necrotizing enterocolitis (17%). At the start of the study, children were getting parenteral nutrition for about six to seven days each week. A stoma was present in 13 (22%) children.
Some children from the 24-week study continued in another 24-week follow-up study and restarted Gattex when their condition worsened or stopped improving.
What Are the Main Benefits Based on the Studies?
Adults
After 24 weeks in the CL0600‑004 and STEPS studies, more adults using Gattex were able to lower the amount of parenteral support they needed by at least 20% compared with those using a placebo. In the STEPS study, more adults receiving Gattex also had a reduction of one or more days per week in their need for parenteral support. At the end of the CL0600‑004 study, two adults treated with Gattex 0.05 mg/kg were able to stop parenteral support completely.
Summary of Outcomes in Adults Treated With Gattex for 24 Weeks
| Gattex 0.10 mg/kg | Gattex 0.05 mg/kg | Placebo | |
|---|---|---|---|
CL0600‑004 study | |||
| Reduction in parenteral support volume of at least 20% | 8/32 (25%) | 16/35 (46%) | 1/16 (6%) |
| No further parenteral support needed | 1/32 (3.1%) | 2/35 (5.7%) | 0/16 (0%) |
STEPS study | |||
| Reduction in parenteral support volume of at least 20% | - | 27/43 (63%) | 13/43 (30%) |
| 1-day or more reduction in the weekly parenteral support | - | 21/39 (54%) | 9/39 (23%) |
| No further parenteral support needed | 0% | 0% | 0% |
In longer follow-up studies, most adults continued to reduce their need for parenteral nutrition while using Gattex.
In the CL0600-005 extension study, 68% (17 out of 25) of the people who used Gattex 0.05 mg/kg/day were able to reduce their weekly parenteral nutrition needs by at least 20%.
In the STEPS-2 extension study, 65 of the 88 people enrolled (74%) completed the study. A total of 29 participants who had previously used a placebo went on to complete 24 months of treatment with Gattex. Of these, 16 people (55%) were able to reduce their parenteral nutrition needs by at least 20%. By the end of the 24-month extension period, 14 participants (48%) reduced their IV nutrition needs by at least one day per week, seven participants (24%) reduced it by at least two days per week, and five participants (17%) reduced it by at least three days per week.
A separate group of 30 participants who had already used Gattex in the STEPS study completed a total of 30 months of Gattex treatment in the STEPS-2 study. Among them, 28 people (93%) were able to reduce their parenteral nutrition needs by at least 20%. By the end of the study, 21 participants (70%) reduced their IV nutrition by at least one day per week, and 18 participants (60%) reduced it by at least three days per week.
In the STEPS-3 study, 13 out of the 14 people enrolled completed the study. Overall, people continued to show improvements in how much parenteral nutrition they needed.
Your results may differ from what was seen in clinical studies.
Children
After 24 weeks, when compared with children who continued standard care, more children who used Gattex were able to reduce their parenteral support volume by at least 20%. Children treated with Gattex also reduced their need for parenteral support by about one day per week. No reduction was seen in children who continued with standard care. Five children who used Gattex were able to stop parenteral support completely.
Summary of Outcomes in Children Treated With Gattex for 24 Weeks
| Gattex 0.025 mg/kg | Gattex 0.05 mg/kg | Standard Care | |
|---|---|---|---|
| Reduction in parenteral support volume of at least 20% | 13/24 (54.2%) | 18/26 (69.2%) | 1/9 (11.1%) |
| Reduction in parenteral nutrition support (days/week) | -0.9 | -1.3 | 0 |
| No further parenteral support needed | 2 (8%) | 3 (12%) | 0 |
In the 24-week follow-up study, most children (87%) needed additional treatment. Over about 40 weeks of total treatment with Gattex, the benefits were similar to those seen after the first 24 weeks. One more child was eventually able to stop parenteral nutrition completely.
In longer follow-up studies lasting almost two years, most children continued to reduce their need for parenteral nutrition while using Gattex.
Your results may differ from what was seen in clinical studies.
How Long Does It Take for Gattex to Work?
People using Gattex usually notice improvements gradually, over several weeks to a few months. In studies with both adults and children who have SBS, many people were able to reduce the amount of parenteral nutrition they needed during the first 24 weeks of treatment with Gattex. Some people continued to make further progress with longer‑term use of Gattex. Gattex has been studied for up to three years in adults and up to two years in children.
How Strong Is the Evidence for Gattex?
Based on current clinical studies, Gattex is safe and effective for adults and children with SBS. Clinical guidelines recommend Gattex for certain people with SBS who need IV nutrition.
How Can I Prevent and Manage Side Effects?
The most common side effects of Gattex include stomach pain, swelling in the stomach area, nausea, and infection in the nose or throat.
Gattex may increase your risk for cancer. Adults should have their colon and upper intestines checked for polyps within six months before starting Gattex. After one year of treatment with Gattex, adults should have their colon and upper intestines checked for polyps again, and thereafter at least every five years. Children should be tested for blood in the stool within six months before starting Gattex. After that, stool testing in children should be done every year. Children should also have their colon checked for polyps after one year of treatment with Gattex, and thereafter every five years.
Keep all appointments for blood tests before starting Gattex and during treatment. Your health care provider will do blood tests to check your gallbladder, liver, and pancreas within six months before starting Gattex. These tests will likely be repeated at least every six months while you are using Gattex. Tell your health care provider right away if you have symptoms of swelling or blockage of your gallbladder or pancreas, such as pain in your stomach, changes in your stool, nausea, vomiting, dark urine, or yellowing of your skin or the whites of your eyes.
Gattex may cause a blockage of your intestines. Tell your health care provider right away if you have symptoms of gut blockage, such as trouble having a bowel movement or passing gas, pain or swelling in the stomach area, nausea, or vomiting.
If the spot where you injected yourself hurts a bit, you can put a warm or cold compress on it to get some relief. Ask your pharmacist or other health care provider whether you can take over-the-counter (OTC) pain medicines to get relief.
These are not all of the possible side effects. Talk with your health care provider if you are having symptoms that bother you. In the U.S., you can report side effects to the FDA at www.fda.gov/medwatch or by calling 800-FDA-1088 (800-332-1088). In Canada, you can report side effects to Health Canada at www.health.gc.ca/medeffect or by calling 866-234-2345.
What Kinds of Drug Interactions May Happen?
Gattex can affect how your body absorbs medicines that you take by mouth. Because Gattex improves the intestine’s ability to absorb nutrients and fluids, it may also increase the absorption of other medicines that you take by mouth. This may change how those medicines work or increase the chance of side effects. For example, Gattex has also been linked with changes in mental status in people taking certain medicines such as benzodiazepines because these medicines may be absorbed more while using Gattex.
Always tell your health care provider about any prescription or OTC medicines, vitamins/minerals, herbal products, and other supplements you are using or have recently taken. This will help make sure your medicines work well together.
Is There Any Cost Assistance Available?
Yes. There is a cost savings and support program offered by the drugmaker, which may allow you to pay as little as $0 for your prescription. Whether you are eligible depends on whether you have prescription insurance and what type of insurance you have. You can find out more information here or by calling 866-888-0660.
