The U.S. Food & Drug Administration (FDA) has approved a treatment for people with amyotrophic lateral sclerosis (ALS) caused by a mutation in the superoxide dismutase 1 (SOD1) gene.
The FDA described Qalsody (also known as tofersen) as “an antisense oligonucleotide that targets SOD1 mRNA to reduce the synthesis of SOD1 protein.”
Qalsody is administered via spinal injection, with a recommended dose of 100 mg per administration. After receiving three doses every 14 days to start, ALS patients receive a maintenance dose every 28 days.
The FDA noted that about 2 percent of people living with ALS in the United States — fewer than 500 individuals — have mutations of the SOD1 gene that lead to so-called SOD1-ALS. The vast majority of ALS cases are considered “sporadic,” meaning there is no family history of ALS and no known associated risk factors.