First batch of children with dwarfism benefit from new drug

The world's first approved medicine to treat an inherited disease causing dwarfism has been introduced by Ruijin-Hainan Hospital in its first new drug program.

Five children with an average age of 5.8 years have become the first batch of patients to benefit from the program.

Ruijin-Hainan Hospital has been involved in a trial project for new drug, equipment introduction and research in China thanks to favorable policies applied to Hainan.

The policies allow the application of medicines not approved in China to be used in Hainan at the Boao Lecheng Pilot Zone of International Medical Tourism, where the hospital is located.

Achondroplasia is a skeletal disorder that starts before birth due to genetic mutation. As cartilage is converted to bone, it results in dwarfism and is the commonest cause of the condition.

It is a rare disease with an incidence of one in every 17,000 to 28,000 people, according to medical experts.

Dr Dong Zhiya, director of pediatrics at Ruijin-Hainan Hospital, said that untreated children with the disease achieve a height of less than 130 centimeters in adulthood and over 80 percent of them suffer other disorders and problems.

These include obesity, hypertension, hydrocephalus and lung disease, seriously impacting their life quality.

Most of the afflicted children's parents are not achondroplasia-gene carriers. The disease stems from the fetus' own genetic mutation, which impacts the normal growth of bone.

The drug Vosoritide, which was marketed in Europe in August 2021, was approved in the US in November. It is the only medicine approved for children with achondroplasia.

Clinical research in the West has proved that patients treated with the drug can achieve much quicker bone growth than untreated patients.

This means the earlier the treatment, the better the results, experts said.