FDA Approves Teduglutide to Treat Short Bowel Syndrome

Mark Crane

Disclosures

December 21, 2012

The US Food and Drug Administration (FDA) today approved teduglutide (Gattex, NPS Pharmaceuticals) to treat the estimated 10,000 to 15,000 American adults with short bowel syndrome (SBS).

Teduglutide, an analogue of GLP-2, a protein involved with intestinal growth and function, is the first targeted therapy for SBS and provides an alternative to intravenous parenteral nutrition. It has been granted orphan drug status by the FDA.

SBS results from the partial or complete surgical removal of the small and/or large intestine. Extensive loss of the small intestine can lead to poor absorption of fluids and nutrients from food.

Teduglutide is an injection administered once daily that helps improve intestinal absorption of fluids and nutrients, reducing the frequency and volume of parenteral nutrition. It is the third FDA-approved drug to treat adults with SBS receiving nutritional support. Somatropin (Zorbtive,EMD Sorono) and glutamine (NutreStore,Emmaus Medical) were approved in 2003 and 2004, respectively.

"Today's approval expands the available treatment options for patients with this life-threatening condition," Victoria Kusiak, MD, deputy director of the Office of Drug Evaluation III in the FDA's Center for Drug Evaluation and Research, said in a press release. "Because Gattex may cause other serious health conditions, it is critical that patients and health care professionals understand the drug's potential and known safety risks."

Patients treated with teduglutide have a potential increased risk of developing cancer and polyps in the intestine, obstructions in the intestine, gallbladder disease, biliary tract disease, and pancreatic disease. To ensure that the benefits of teduglutide outweigh the potential risks, the drug is being approved with a Risk Evaluation and Mitigation Strategy (REMS), consisting of a communication plan and training for prescribers, FDA said.

"The relationship with malignancy cannot be completely ruled out at this time, and we suggest warning patients that Gattex may accelerate the growth of malignancy and increase the risk of malignancy," John Troiani, MD, PhD, from the FDA's Center for Drug Evaluation and Research, said in October when the FDA Gastrointestinal Drugs Advisory Committee voted unanimously that teduglutide should be approved.

Teduglutide was evaluated in 2 clinical trials and 2 extension studies. The clinical trials were designed to measure the number of patients who achieved at least 20% reduction in the volume of weekly parenteral nutrition after 20 and 24 weeks of treatment (clinical response). Forty-six percent and 63% of patients treated with teduglutide achieved clinical response vs 6% and 30% of patients treated with placebo.

The trials also showed a mean reduction in parenteral nutrition of 2.5 L/week and 4.4 L/week in teduglutide-treated patients compared with 0.9 L/week and 2.3 L/week in placebo-treated patients.

The most common adverse effects of teduglutide identified in clinical trials were abdominal pain, injection site reactions, nausea, headaches, abdominal distension, and upper respiratory tract infection.

To study teduglutide's long-term safety, the FDA is requiring a postmarketing study of patients with SBS treated with the drug in a routine clinical setting to further evaluate the drug's potential increased risk to cause colorectal cancer and other conditions. Patients in this study will be followed for at least 10 years.

Gattex is marketed by NPS Pharmaceuticals, Bedminster, New Jersey.

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