In a landmark development for neurological care, pharmaceutical company Biogen has commercially launched Tofersen – the first gene-targeted therapy for amyotrophic lateral sclerosis (ALS) caused by SOD1 gene mutations – in the Chinese mainland this week. The drug's arrival marks a turning point in treating this rapidly progressive neurodegenerative disease.
A Lifeline Against 'Grave Stealer' Disease
Known colloquially as the 'grave stealer' for its devastating progression, ALS destroys motor neurons controlling voluntary muscles. Most patients face respiratory failure within 3-5 years of diagnosis. Tofersen specifically targets SOD1-ALS, which accounts for a significant portion of cases in China and typically emerges around age 50.
Science Meets Urgent Need
The antisense oligonucleotide drug works by inhibiting production of toxic SOD1 proteins, slowing neuron damage. Its first administration at Peking University Third Hospital symbolizes new clinical possibilities. 'This addresses a critical unmet need,' said a hospital neurologist involved in the treatment.
Broader Implications
While currently focused on SOD1-ALS patients, Tofersen's success could accelerate research into genetic therapies for other neurodegenerative conditions. The launch also highlights China's growing role in adopting cutting-edge medical treatments, with analysts noting potential market growth in precision neurology.
