Gattex

Indications for Use

Gattex is specifically approved for the treatment of short bowel syndrome (SBS). This condition occurs when the body cannot absorb enough nutrients or fluids because a large portion of the small intestine is missing or not functioning correctly, often due to surgery or injury.

Patient Population

This medication is used for:

• Adults with short bowel syndrome.
• Children and infants 1 year of age and older with short bowel syndrome.

Treatment Goals

Gattex is prescribed for patients who are dependent on parenteral support, which means they receive nutrition or fluids through an intravenous (IV) line. The primary goal of treatment is to improve the absorption of nutrients and fluids from the remaining intestine. Over time, this may help reduce the volume or frequency of IV support required by the patient.

Mechanism of Action

Gattex is a synthetic version of a hormone called glucagon-like peptide-2 (GLP-2). This hormone is naturally produced in the body and is responsible for maintaining the health and function of the intestinal lining.

In people with short bowel syndrome, the body often struggles to absorb enough fluids and nutrients because a significant portion of the small intestine is missing or damaged. Gattex helps the remaining part of the intestine work more effectively through several processes:

• Increasing Surface Area: It helps grow the small, finger-like projections called villi that line the intestine, which increases the area available for absorption.
• Improving Absorption: It enhances the body's ability to take in water, electrolytes, and nutrients from food and drinks.
• Slowing Digestion: It slows down the movement of food through the digestive tract, providing more time for the intestine to absorb what is needed.

By improving the intestine's ability to absorb nutrients, Gattex may reduce the volume of parenteral nutrition (IV feeding) or fluids a person requires to stay healthy.

Common Side Effects

Many people using Gattex experience mild to moderate side effects. These often include:

• Stomach pain, bloating, or gas
• Nausea and vomiting
• Swelling in the hands, ankles, or feet
• Redness, pain, or swelling where the medicine was injected
• Headache
• Cold symptoms, such as a sore throat or stuffy nose

Serious Side Effects

While less common, some side effects can be severe and require medical attention. These include:

• Intestinal obstruction: A blockage in the bowel that can cause severe pain and vomiting.
• Gallbladder and pancreas problems: Symptoms may include pain in the upper right or middle stomach area, fever, or yellowing of the skin and eyes.
• Fluid overload: Excessive fluid buildup that may cause sudden weight gain or difficulty breathing.
• Growth of polyps or tumors: Because Gattex promotes cell growth in the intestines, it may increase the risk of abnormal growths or cancer in the digestive tract.

Risk of Cancer and Polyps

Gattex may promote the growth of abnormal cells, including polyps in the colon or cancer. A colonoscopy is typically required before starting treatment and periodically thereafter. If polyps are discovered, they should be removed; if a malignancy is found, the medication is typically stopped.

Gallbladder and Pancreas Complications

Treatment can lead to problems with the gallbladder, bile ducts, or pancreas, such as stones or inflammation. Monitoring often includes blood tests for lipase, amylase, bilirubin, and alkaline phosphatase. Symptoms like abdominal pain, nausea, or jaundice require medical evaluation.

Intestinal Obstruction

There is a potential for the bowel to become blocked. Individuals should be monitored for signs of intestinal obstruction, such as severe abdominal pain, swelling, or vomiting.

Fluid Overload

This medication can cause the body to retain excess fluid, which may worsen heart failure or cause swelling and high blood pressure. Regular monitoring of weight and physical signs of fluid retention is recommended.

Increased Drug Absorption

Gattex increases the absorption of nutrients and may also increase the absorption of other oral medications. This can lead to higher-than-intended levels of other drugs in the system, potentially requiring dosage adjustments for those medications.

Pregnancy

There are no adequate and well-controlled studies of Gattex in pregnant women to determine if there is a drug-associated risk of major birth defects or miscarriage. Animal reproduction studies using doses significantly higher than the recommended human dose did not show evidence of harm to the developing fetus. However, because animal studies do not always predict human response, Gattex should be used during pregnancy only if the potential benefit justifies the potential risk.

If you are pregnant or planning to become pregnant, consult your healthcare provider to discuss your treatment options and the potential impact on the pregnancy.

Breastfeeding

It is not known whether Gattex passes into human breast milk or how it might affect a nursing infant or milk production. Because many drugs are excreted in human milk and there is a potential for serious adverse reactions in nursing infants, a decision should be made whether to discontinue breastfeeding or discontinue the drug.

Healthcare providers typically weigh the importance of the medication to the mother against the potential risks to the infant when making a recommendation. It is important to talk with your doctor about the best way to feed your baby while using this medication.

Available Forms

Gattex is supplied as a sterile, preservative-free powder in a single-dose glass vial. It is administered by subcutaneous injection.

The medication is typically available in a kit that includes the following components for preparation and administration:

• Vials containing the medication powder
• Prefilled syringes containing sterile water for injection, which serves as the diluent
• Disposable needles and syringes
• Alcohol swabs

Before use, the powder is reconstituted with the provided liquid to form a solution. The resulting liquid should be clear and colorless to pale yellow, without any visible particles.

FDA Approval History

The U.S. Food and Drug Administration (FDA) first approved Gattex in December 2012 for adults with short bowel syndrome who depend on parenteral support. In May 2019, the FDA expanded this approval to include pediatric patients aged 1 year and older.

Risk Evaluation and Mitigation Strategy (REMS)

Gattex is subject to a Risk Evaluation and Mitigation Strategy (REMS) program. This safety program is required by the FDA to ensure that the benefits of the drug outweigh the risks of potential side effects, specifically the risk of accelerated neoplastic growth (abnormal cell growth) and the development of colorectal polyps.

Generic Availability

Currently, there is no FDA-approved generic version of Gattex available in the United States. It is classified as an orphan drug, which is a status granted to medications intended for the treatment of rare diseases or conditions.

Factors Influencing Cost

The price of Gattex varies significantly based on several factors, including the specific insurance plan, the pharmacy used, and the dosage required. Because it is a specialty medication, it is typically not available at standard retail pharmacies and must be ordered through a specialty pharmacy.

Insurance Coverage

Most private insurance plans and Medicare Part D provide coverage for Gattex, though it often falls into a high-cost specialty tier. This may result in significant out-of-pocket expenses, such as deductibles or coinsurance payments. Prior authorization from a healthcare provider is usually required before insurance will approve coverage.

Generic Availability

There is currently no generic version of Gattex (teduglutide) available in the United States. Brand-name specialty drugs generally remain expensive until a generic alternative is approved by the FDA and released to the market.

Financial Assistance

The manufacturer of Gattex may offer programs to help reduce the cost for eligible patients. These options often include:

• Copay Assistance Cards: These can help patients with commercial insurance lower their out-of-pocket costs.
• Patient Assistance Programs: These programs may provide the medication at no cost or a reduced cost for individuals who meet specific income requirements and lack adequate insurance coverage.