The Japanese Ministry of Health, Labor and Welfare (MHLW) has granted orphan drug designation to NPS Pharmaceuticals' teduglutide for the treatment of Short Bowel Syndrome (SBS).

Also, due to the strong medical need for SBS treatments in Japan, the MHLW's expert committee on Unapproved New Drugs and New Indications with High Medical Needs requested that NPS Pharma Japan K.K. develop teduglutide for adult SBS earlier this year. To meet local regulatory requirements, the company is also preparing to initiate a small study in Japanese patients with SBS before the end of this year.

"The decision to grant teduglutide orphan drug status in Japan underscores the need for new treatment options for people living with Short Bowel Syndrome," said Roger Garceau, MD, executive vice president and chief medical officer of NPS Pharma. "We look forward to initiating our study and advancing the regulatory process for teduglutide in Japan."

In Japan, orphan drug status can be bestowed on drugs or medical devices intended for use in fewer than 50,000 patients for which there is a high medical need. Orphan drugs may be eligible for subsidies and tax credits to reduce the burden of product development, priority consultation and review for marketing authorization, and an extended reexamination period of up to 10 years.

The MHLW established the committee on Unapproved New Drugs and New Indications with High Medical Needs to enhance the development of drugs and indications that have been approved for use in the United States and Europe, but are not yet approved in Japan. Due to the strong medical need for SBS treatments in Japan, the committee determined that teduglutide met these criteria and requested that the company develop the drug for adult SBS.

Teduglutide received orphan drug designation for the treatment of SBS from the US Food and Drug Administration in 2000 and the European Medicines Agency in 2001. In the US, NPS Pharma markets teduglutide under the trade name Gattex and in the EU, as Revestive.

Short Bowel Syndrome, or SBS, is a rare and potentially fatal gastrointestinal disorder in which patients are unable to absorb enough nutrients and fluids through the intestine from the food they eat to sustain life. SBS typically occurs when a large portion of the intestine has been removed by surgery caused by disease or injury. In rare cases, it is congenital. Common symptoms of SBS can include diarrhea, dehydration, malnutrition, and weight loss, which are closely related to the functional issues of the bowel. Patients with SBS may be dependent on parenteral nutrition, a nutritional supplement administered intravenously, for their survival.