CIIE effect benefits innovative medicine for rare disease sufferers

Cai Wenjun
New targeted therapy against short bowel syndrome used to treat five child patients at Xinhua Hospital to reduce their dependence on invasive procedures.
Cai Wenjun

Benefiting from the spillover effects of the China International Import Expo, an innovative medicine was approved by the Chinese authority ahead of World Rare Disease Day next Thursday.

The innovative medicine teduglutide is allowed to be used in Hainan Province's Bo'ao six months after its 2022 CIIE debut, as Bo'ao is allowed for medicines yet to be approved in China to carry out clinical trials. The process facilitates teduglutide receiving an approval.

It is the first targeted therapy for patients with short bowel syndrome, a disease affecting an extremely small quantity of patients – about 0.73 among every 1 million adults in the nation with a rising tendency.

Short bowel syndrome is a condition in which the body cannot absorb enough nutrients from food because part of the small intestine is missing or damaged. The small intestine is where most nutrients are absorbed during digestion.

Due to a lack of effective medicines, domestic patients with short bowel syndrome have to depend on parenteral nutrition (nutrition delivered through a tube to the stomach or small intestine) and intravenous injections. However long-term treatment can cause serious and even fatal complications. The economic burden is also high.

Teduglutide, developed by Takeda, is a glucagon-like peptide 2 (GLP-2) analogue to help patients reduce the need for parenteral support. It leads to the growth of intestinal mucosa by stimulating intestinal crypt cell growth and inhibiting enterocyte apoptosis.

"Patients desperately want to have medicines which can help them reduce dependence on parenteral nutrition. We are very glad to see the approval of this innovative medicine, which gives us a new weapon against the disease," said Dr Cai Wei from Xinhua Hospital, whose five child patients suffering from the disease took part in the clinical trial in Bo'ao.

CIIE effect benefits innovative medicine for rare disease sufferers
Ti Gong

A child with short bowel syndrome receives a teduglutide injection.

"The five children have shown positive effects after receiving a teduglutide injection. We are eager to see more patients benefit from the innovative medicine after its official approval," Cai said.

Diseases with incidences of 0.65 to 1 per 1,000 are identified as rare diseases. There are over 7,000 rare diseases worldwide. About 80 percent are inherited, affecting some 350 million people globally and over 20 million in China. About half of rare disease sufferers are children. About 30 percent of children with rare diseases die before the age of five. Rare diseases are also responsible for 35 percent of infant deaths younger than one year old.

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