'Wonder drug' treats boy in city hospital
A boy with Fabry disease is injected with agalsidase beta at the Children's Hospital of Fudan University on Thursday.
A child with Fabry disease was injected with a so-called wonder drug — agalsidase beta — at the Children's Hospital of Fudan University on Thursday.
Fabry disease is a rare condition related to gene mutation. In childhood and adolescence, patients will suffer nerve pain and excessive sweating or an inability to sweat. Complications include kidney failure and apoplexy, which can cause their premature death.
Because of non-specific symptoms in the early stage and a lack of understanding of the disease, it can be many years before the disease is diagnosed.
So that more children be diagnosed and treated early, a multidisciplinary team was established in April by the members of the national panel for Fabry disease and Professor Xu Hong from the Children's Hospital of Fudan University.
So far, 20 children have been screened by the team, three are waiting for the genetic test results and one had been diagnosed. The boy, from Jiangsu Province, is the first diagnosed patient in China.
Agalsidase beta was approved by the national medical products watchdog in December last year.
Agalsidase beta was approved by the national medical products watchdog last year.
The cost of the drug is not covered by the city’s medical insurance system but is included on the medical insurance list in neighboring Zhejiang Province. Professor Lu Jun, an expert with the National Health Commission, said if it can be covered by the local medical insurance system, more children suffering from Fabry disease can be treated.
An earlier report said the cost of 5 milligrams is 8,995.35 yuan (US$1,300) and the injection volume depends on the weight of the patient. A 59.5-kilogram adult and a 33kg child could spend 1.98 million yuan and 990,000 yuan, respectively, each year for the drug.
Xu said the incidence of the disease is likely to be underestimated based on the screening data and results.
According to this year’s statistics from Chinese Organizations for Rare Disorders and Pharmaceutical consultancy IQVIA, the estimated number of Fabry disease cases in China is about 18,860, but only 366 cases have been registered.
