FDA greenlights Biogen's Qalsody for rare, genetic form of ALS

The US Food and Drug Administration (FDA) yesterday approved Qalsody (tofersen) to treat patients with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS).

Developed by US biotech Biogen (Nasdaq: BIIB), Qalsody is an antisense oligonucleotide that targets SOD1 mRNA to reduce the synthesis of SOD1 protein. The approval was based on a reduction in plasma neurofilament light (NfL), a blood-based biomarker of axonal (nerve) injury and neurodegeneration.

This indication is approved under accelerated approval based on reduction in plasma neurofilament light chain (NfL) observed in patients treated with Qalsody. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s). The ongoing Phase III ATLAS study of tofersen in people with presymptomatic SOD1-ALS will serve as the confirmatory trial, said Biogen.