Biogen’s Qalsody posts long-term signal in rare genetic ALS

US biotech Biogen (Nasdaq: BIIB) said final Phase III data published in JAMA Neurology support earlier use of Qalsody (tofersen) in patients with superoxide dismutase 1 amyotrophic lateral sclerosis, pointing to slower decline across function, breathing and strength with extended follow-up.

Results from the completed VALOR study and its open-label extension, with more than 3.5 years of follow-up, also suggested a lower risk of death or permanent ventilation when treatment was started earlier. Sustained reductions in neurofilament supported an effect on the underlying disease process.

Qalsody is approved in 44 countries. In the USA, the drug received accelerated approval from the American medicines regulator based on reductions in plasma neurofilament, with continued approval dependent on confirmation of clinical benefit. Safety findings were consistent with earlier reports.