Breakthrough gene-targeted drug for ALS patients launched in China

Tofersen, a breakthrough gene-targeted drug for amyotrophic lateral sclerosis (ALS) patients, was commercially launched by pharmaceutical giant Biogen in China on Tuesday, marking a key step toward treating this fatal disease.

ALS progressively destroys motor neurons in the brain and spinal cord, leading to muscle weakness, atrophy and typically fatal respiratory failure within three to five years.

The first injection was administered at Peking University Third Hospital, signifying the clinical availability of China's first therapy targeting ALS caused by superoxide dismutase 1 (SOD1) gene mutations (SOD1-ALS).

SOD1 is the first identified ALS-causing gene and the most common pathogenic gene among Chinese ALS patients. SOD1-ALS often manifests around age 50, starting in the limbs with combined upper and lower motor neuron damage. 

Tofersen, an antisense oligonucleotide (ASO), works by reducing the synthesis of toxic SOD1 protein, thereby slowing motor neuron damage and disease progression.

This precision medicine for ALS was approved in China in September 2024. Its commercial launch addresses a critical need and pioneers a new era of precision diagnosis and treatment for ALS in China, driven by national rare disease strategies and policies accelerating innovative drug access.  

"For ALS, especially SOD1-ALS, early detection, diagnosis and treatment are paramount," said Professor Fan Dongsheng from the Department of Neurology at Peking University Third Hospital. 

"The drug's launch is a landmark, opening China's era of targeted ALS therapy. We urge standardized genetic testing and diagnosis to help eligible patients access this breakthrough during the crucial treatment window," Fan said.

Wang Jinhuan, chairman of the Beijing Oriental Rain ALS Care Center, expressed patient community excitement. 

"SOD1-ALS patients have long lacked effective treatments. The drug ignites hope. We deeply appreciate national policy support, Biogen's scientific commitment and collaborative efforts bringing this innovation to China," said Wang.

Ding Weibo, head of Biogen's Asia Pacific region, said that the drug's launch is a major milestone in the fight against ALS.

"Its launch in China is made possible by supportive policies, efficient regulatory review and collaboration with clinicians and patient groups," Ding said.