IFFGD Vice-President, William Norton, submitted the following written public testimony to the FDA in connection with an October 16, 2012 meeting of the Gastrointestinal Drugs Advisory Committee to consider the new drug application (NDA) 203441 by NPS Pharmaceuticals for the drug with the proposed trade name GATTEX® (teduglutide) for the treatment of adult patients with short bowel syndrome (SBS):

On behalf of the International Foundation for Functional Gastrointestinal Disorders (IFFGD), I thank the Food and Drug Administration (FDA) for its work to evaluate treatments for conditions that affect the public. IFFGD is a 501(c)(3) nonprofit education and research organization dedicated to improving the understanding of functional gastrointestinal and motility disorders.

I am writing to you to offer comments regarding the October 16, 2012 meeting of the Gastrointestinal Drugs Advisory Committee. At this meeting, the Advisory Committee will consider the new drug application (NDA) 203441 by NPS Pharmaceuticals for the drug with the proposed trade name GATTEX® (teduglutide) for the treatment of adult patients with short bowel syndrome (SBS).

Short bowel syndrome is a complex condition characterized by the loss of absorptive capacity of the small intestine. It can occur in people at any age. The cause can be congenital, or acquired due to a variety of reasons. In some the condition can be life-threatening.

People face numerous difficulties as a result of having SBS. In addition to managing multiple symptoms, extraordinary measures must be taken to ensure that individual fluid and nutrient requirements are met. These measures include high levels of doctor-patient communication and patient/care-giver education, frequent healthcare utilization, carefully monitored diets, and often the need to use parenteral nutrition and intravenous fluids. The long-term use of parenteral support increases risks of infection and other complications, which themselves can be life-threatening, and is associated with numerous quality of life issues such as loss of sleep, mobility, and social interactions.

Treatment options over the long course of SBS are burdensome on patients, care-providers, and healthcare providers. Treatments are aimed primarily at maintaining adequate nutritional status and preventing complications. However, complications can arise not only as a result of the underlying condition, but also in connection with treatments such as parenteral infusions.

New treatment options are needed to help people with short bowel syndrome more effectively manage their condition, maintain required nutritional status, and reduce use of parenteral support for those who rely on it. If Gattex® (teduglutide) is found to be a safe and effective therapy, it would be a significant step forward for patients with short bowel syndrome.

Thank you for your consideration of our comments.