QALSODY (tofersen) is an antisense oligonucleotide designed to treat amyotrophic lateral sclerosis (ALS) in adults with a specific mutation in the superoxide dismutase 1 (SOD1) gene. Its therapeutic effect is based on a reduction of plasma neurofilament light chain levels, which is linked to neurodegeneration in ALS.

Therapeutic Mechanism and Efficacy of QALSODY in ALS Treatment

QALSODY (tofersen) is indicated for the treatment of ALS in patients with mutations in the SOD1 gene. Its mechanism of action involves the reduction of neurofilament light chain (NfL) levels in plasma, a biomarker for neuronal damage. The reduction in NfL has been associated with potential clinical benefits in ALS management. The drug's approval was based on accelerated clinical evidence, specifically the decrease in NfL, though continued approval is contingent on further verification of clinical efficacy in confirmatory trials. This provides a promising avenue for ALS patients with SOD1 mutations, potentially slowing disease progression.