2023-07-30
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.