In February, Li Kegiang, Premier of the State Council, announced that the Chinese government was going to take steps to improve rare disease patients access to medicine. As part of this initiative, Spinraza, the only approved treatment for spinal muscular atrophy (SMA), has just entered the Chinese market.
Rare Disease in China
There are more than 20 million people living with rare diseases in China. The average medical costs per year for these individuals is 200,000 yuan. That’s 29,700 dollars. 40% of rare disease patients in the country receive no treatment due to the high costs.
There are currently 55 different rare disease drugs available to patients in China. Of these, China’s medical insurance system covers 29 in part or in full.
These numbers come from the China Alliance of Rare Diseases.
SMA specifically affects approximately one out of every 6,000-10,000 individuals. Currently, 95% of these patients don’t live longer than 18 months.
What exactly is SMA? SMA is a rare condition that results in muscle degeneration and muscle weakness. There are four different types of the disease, which vary by severity and age of onset.
SMA is caused by mutations in the SMN1 gene. Patients experience a loss of motor neurons in the brainstem and spinal cord. They may face difficulties breathing, swallowing, walking, moving their head, or even sitting up. Other symptoms may include tremors or developmental delays.
Spinraza is the first therapy approved to treat SMA. Spinraza was first approved in the United States in 2016. It is currently available in over 40 different countries/regions. Before its approval (and in countries where it has not yet entered the market), patients relied on supportive care. Patients may receive physical therapy, braces, a tracheostomy, gastrostomy, noninvasive respiratory support, and/or surgery.
Up until now, SMA patients in China have survived on this symptomatic treatment.
Spinraza in China
The process to get Spinraza approved in China began back in September and it was fast tracked through the process of market approval.
Biogen, who developed Spinraza, has announced that they are in the process of working with China’s government, nonprofits, and the patient community to establish a copayment system for the drug. They hope that this system will help to make it more accessible for SMA patients.
You can read more about this new development here.