Rare disease drug included in medical insurance list

A rare disease drug was officially listed in national medical insurance, according to a release by the National Healthcare Security Administration and the Ministry of Human Resources and Social Security published on Wednesday.

Evrysdi, or risdiplam, made by Roche is to treat spinal muscular atrophy (SMA) patients above two-month-old and was approved in June of 2021 to become the nation's first-adopted SMA oral drug.

SMA is one of the rare diseases of the neurological system and can cause disability or death. China witnesses about 1,000 newborns with the disease per year. The disease causes malfunctions of the bone, respiratory and other systems and eventually respiratory failure within one year after birth.

Other SMA patients are diagnosed during their teen years or adulthood and have already suffered other neurological or muscle diseases.

Patients don't need operations nor hospitalization if taking this oral drug and infant patients are relieved from taking multi drugs.

Wang Yi, professor of Children's Hospital affiliated with Fudan University in Shanghai, said listing Evrysdi in medical insurance is an important supplement to clinical treatment and will bring hope and new life to SME patients.

SMA was included in the nation's rare diseases list in 2018 and Evrysdi was preferentially taken into the procedure to be approved.