China has taken a monumental step in the fight against amyotrophic lateral sclerosis (ALS) with the commercial launch of Tofersen, a groundbreaking gene-targeted drug.
On Tuesday, pharmaceutical giant Biogen introduced Tofersen to the Chinese market, offering new hope to patients battling this fatal neurological disease. The first injection was administered at Peking University Third Hospital, marking the drug’s official entry into clinical use.
ALS, often known as Lou Gehrig’s disease, progressively destroys motor neurons in the brain and spinal cord. This leads to muscle weakness, atrophy, and ultimately fatal respiratory failure, typically within three to five years of onset.
Tofersen specifically targets ALS caused by mutations in the superoxide dismutase 1 (SOD1) gene, referred to as SOD1-ALS. SOD1 is not only the first identified gene causing ALS but also the most common pathogenic gene among Chinese ALS patients.
As an antisense oligonucleotide (ASO), Tofersen works by reducing the production of the toxic SOD1 protein, thereby slowing the damage to motor neurons and the progression of the disease.
“The launch of Tofersen is a landmark moment, opening a new era of targeted therapy for ALS in China,” said Professor Fan Dongsheng from the Department of Neurology at Peking University Third Hospital. “Early detection, diagnosis, and treatment are paramount for patients with SOD1-ALS. We urge standardized genetic testing to help eligible patients access this breakthrough during the crucial treatment window.”
The approval of Tofersen in China addresses a critical unmet need and pioneers a new era of precision diagnosis and treatment for ALS in the country. This advancement is driven by national rare disease strategies and policies that accelerate the availability of innovative drugs.
Wang Jinhuan, chairman of the Beijing Oriental Rain ALS Care Center, expressed the patient community’s excitement. “SOD1-ALS patients have long lacked effective treatments. The introduction of Tofersen ignites hope within the community. We deeply appreciate the national policy support, Biogen’s scientific commitment, and the collaborative efforts that brought this innovation to China,” Wang said.
Ding Weibo, head of Biogen’s Asia Pacific region, emphasized the collaborative efforts behind the drug’s launch. “Tofersen’s availability in China is a major milestone in the fight against ALS. This achievement was made possible by supportive policies, efficient regulatory review, and cooperation with clinicians and patient groups,” Ding remarked.
The introduction of Tofersen in China not only offers a lifeline to patients but also sets a precedent for future advancements in treating rare diseases. As the medical community continues to push boundaries, patients and families affected by ALS can find renewed hope for the future.
