One injection of 13.5 million yuan may cure SMA, the most expensive drug in history will land in China

Yangcheng Evening News reporter Zhang Hua

Recently, the Center for Drug Evaluation (CDE) of China's State Food and Drug Administration announced that Novartis' AAV gene therapy drug Zolgensma (OAV101 injection) for the treatment of Spinal Muscular Atrophy (hereinafter referred to as "SMA") Drug clinical trial applications submitted in China have obtained implied permission for clinical trials

One shot for long-term relief or cure

SMA is an inherited neuromuscular disorder that causes muscle weakness and atrophy due to degeneration of cells in the anterior horn of the spinal cord

According to statistics, the incidence of neonatal SMA is about 1/10000

At present, the SMA treatment drug included in China's medical insurance is Spinraza (Nocinnagen Sodium Injection)

In fact, there is a cure for SMA

Liu Li, chief physician of the Department of Genetics and Endocrinology of Guangzhou Women's and Children's Medical Center, chairman of the Rare Disease Branch of Guangdong Medical Association, and project leader of the Guangdong Rare Disease Diagnosis and Treatment Collaboration Network, introduced to reporters that Chinese children use drugs abroad From the results, the one-time drug treatment is of great help in improving the symptoms of children, but it is extremely expensive

A total of three drugs have been approved worldwide

Because it is a one-shot cure, the price of Zolgensma is much higher than that of Nosinagen Sodium Injection.

It is the preliminary work that needs to be done before the drug is officially submitted for approval and marketing to clarify the safety and effectiveness of the drug for patients in China through drug clinical trials

According to the reporter's understanding, there are currently three SMA therapies approved in the world, namely Zolgensma from Novartis, Spinraza (Nosinagen Sodium Injection) from Biogen, and Risdiplam (Risprelam) from Roche

Liu Li said that if Zolgensma enters China, there will be an additional option for SMA patients