US company partners with Chinese firm to tackle rare disease

SHANGHAI — Staff with US company Amicus Therapeutics regularly travel to Shanghai to work out how to develop a new drug to treat Pompe disease, a rare muscle disorder.

John Crowley, chief executive of the company, led the recent trip to its Chinese partner WuXi Biologics, based in Shanghai.

Crowley said the new drug was expected to reduce the side effects of existing enzyme replacement therapy.

The disorder prevents the human body from making enough of an enzyme used by heart and muscle cells to convert a form of sugar called glycogen into energy. When glycogen builds up in these cells it can lead to swelling of the heart, disability and early death.

Crowley has led a personal struggle to save his daughter and son, who are afflicted with Pompe disease.

Crowley said his company chose WuXi Biologics as its contract manufacturer, even though some company board members questioned the decision, doubting such high-quality drugs could be made by a Chinese firm.

Currently, the drug is in stage two of clinical trials, and around 20 patients are being tested with the drug in the United States.

By the middle of next year, it is expected to enter stage three, and be applied to more patients for tests, Crowley said.

Chinese patients are expected to be included in the trials, he said. Gao Ru, founder of a Chinese Pompe patient association, said the cooperation would benefit Chinese patients. She estimated there are about 35,000 Pompe patients in China.

Gao said she managed to contact about 100 of them, 80 percent of whom were adults, and 20 percent babies.

For an adult patient, the annual medical cost is around 3 million yuan (about $460,000).

"I hope the cost for the prescription can be reduced," she said.

Crowley helped founded Novazyme, a company working on Pompe disease treatment. The company was was acquired by Genzyme in 2011. Now Paris-based Sanofi SA owns the drugs. He later founded Amicus Therapeutics to develop therapy for rare disease including Pompe.

"If you compare ordinary drugs to a bicycle, bio-medicine is like a plane. It is very difficult to make," said Chen Zhisheng, CEO of WuXi Biologics.

"The new generation drugs to treat Pompe diseases needs to be very accurate in dealing with the disorder. Both our companies will be committed to the development of the drugs until they are put on the market and bring benefits to patients," Chen said.