FDA grants accelerated approval for Qalsody for SOD1-ALS, a major scientific advancement as the first treatment to target a genetic cause of ALS

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.

Qalsody is the first approved treatment to target a genetic cause of ALS. Biogen collaborated with Ionis Pharmaceuticals on the early development of tofersen.

Warnings and precautions associated with Qalsody were serious neurologic events, including myelitis and/or radiculitis; papilledema and elevated intracranial pressure; and aseptic meningitis. If symptoms consistent with myelitis, radiculitis papilledema, elevated intracranial, or aseptic meningitis develop, diagnostic workup and treatment should be initiated according to the standard of care. Management may require interruption or discontinuation of Qalsody The most common adverse reactions that occurred in greater than 10% of Qalsody treated participants and more than the placebo arm were pain, fatigue, arthralgia, cerebrospinal (CSF) white blood cell increased, and myalgia.