Biogen Receives FDA Approval for Qalsody in SOD1-ALS

Biogen today announced that the FDA has approved Qalsody (tofersen) for the treatment of adults with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene. Qalsody is an antisense oligonucleotide (ASO) that reduces the production of SOD1 protein. In the VALOR trial, Qalsody demonstrated a statistically significant reduction in neurofilament light chain (NfL) levels, a biomarker of neurodegeneration. While the primary clinical endpoint was not met in the 28-week trial, longer-term data showed clinical benefits in patients who started treatment earlier. This approval represents the first targeted treatment for a genetic form of ALS.