Gattex (ingredient name: Teduglutide), Shire’s short bowel syndrome (SBS) treatment, could bring a glimmer of hope upon young patients, as it received sales approval from the Ministry of Food and Drug Safety, the company said.
|Professor Lee Sang-hoon of the department of pediatrics at Samsung Medical Center explains the beneficial aspects of Gattex, during a media conference at Lotte Hotel Seoul in Myeongdong, Tuesday.|
The drug is the first approved treatment for SBS in Korea. SBS occurs in patients older than one year. Congenital or postoperative surgical resection causes more than 50 percent of the total small intestine to disappear, causing malabsorption and malnutrition.
The disease has received so little attention that the government does not have an exact number of patients suffering from the illness. Given the global prevalence rate is 24.5 per 100,000 people, however, the number of patients in Korea is expected to be about 12,000.
Until now, patients suffering from SBS had to receive a daily intravenous injection of total parenteral nutrition (TPN) as they did not have enough small intestines involved in digestion and absorption of food.
However, TPN has a risk of causing sepsis and thrombosis due to infection of the inserter and insertion site. The method also takes up 10-hours a day, leaving little time for patients to live a normal life.
There are surgical options that can improve bowel absorption such as small intestine transplant and serial transverse enteroplasty (STEP), which is a procedure that makes cuts in the intestine and creating a zigzag pattern to lengthen the amount of bowel available to absorb nutrients.
However, it is difficult to recognize such operations as complete remedies. In the case of a small intestine transplant, there have been only 25 cases operated in Korea so far.
“Small intestinal transplantation is the least active of all transplants,” said Professor Lee Sang-hoon of the department of pediatrics at Samsung Medical Center. “It may be because there are only a few patients, but the results of the surgery are not very good.”
Especially in children, surgery is a little more difficult as it is difficult to find suitable donors, Professor Lee added.
Regarding STEP, Lee stressed, even though the operation is a more viable option, it is not a complete cure as the TPN injection may continue after the surgery.
In this regard, Gattex can drastically improve quality of life as patients only need to inject the treatment into their thighs, arms or abdomen once a day.
Glucagon-like peptide-2 (GLP-2) formulation, which is the main component of Gattex, is also advantageous for patients. In the case of short bowel movements like short-bowel syndrome, GLP-2 enlarges the villi in the small intestine and the body surface area, thus enhancing the ability to absorb nutrients.
The company said it has already proved the drug’s safety and efficacy through a phase 3 clinical trial.
The trial results showed that during week 20 to 24 Gattex reduced the dose of TPN by 20 percent in 27 out of the 43 patients that participated in the research. During the same period, only 13 out of 43 patients in the placebo group managed to reduce TPN doses.
Also, 48.8 percent of patients, who had completed the study, had improved so much that they no longer needed the TPN treatment.
“The ultimate goal of treatment for pediatric patients and their parents is to stop TPN injections,” Professor Lee said. “In the past, there had been similar medicines that have tried to resolve this issue, but no medicines showed clinically meaningful results.”
As Gattex has proved its safety and efficacy it will become a new hope for patients and their families in treating SBS, Lee added.
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