On July 14, Bojian's listing application for tofersen injection was accepted.
On April 25, 2023, the FDA accelerated approval of the antisense oligonucleotide therapy tofersen (Qalsody) for the treatment of amyotrophic lateral sclerosis (ALS) in patients with superoxide dismutase 1 (SOD1) mutations. This is the first gene-targeted therapy for ALS.
ALS, commonly known as ALS, is a progressive neurodegenerative disease characterized by progressive skeletal muscle atrophy, weakness, and fascicular fibrillation. The average survival time of patients is 3 to 5 years. The most common cause of death is respiratory failure. SOD1-ALS is a rare genetic form of ALS, accounting for only 2% of the approximately 168,000 ALS cases worldwide, most patients with rapid disease progression, survival after onset of less than three years.