Long-Term Tofersen in Amyotrophic Lateral Sclerosis

What are the long-term effects of early-start and placebo/delayed-start tofersen treatment in adults with amyotrophic lateral sclerosis (ALS)?This integrated analysis of the phase 3, randomized, double-blind, placebo-controlled VALOR study and its open-label extension (OLE) found that participants with earlier initiation of tofersen exhibited numerically less decline in function, strength, and risk of death-equivalent events than participants in the placebo/delayed-start group (tofersen initiated approximately 6 months later), consistent with a slowing of disease progression. No new safety concerns were identified with long-term exposure to tofersen.Final data from VALOR and its OLE demonstrated the benefit of tofersen treatment for adults with -ALS and provide a clear rationale for starting therapy in this population.

Corresponding Authors: Timothy M. Miller, MD, Department of Neurology, Neuromuscular Medicine, Washington University School of Medicine in St Louis, 660 S Euclid Ave, Box 8111, St Louis, MO 63110 (millert@neuro.wustl.edu); Stephanie Fradette, PharmD, Biogen, 225 Binney St, Cambridge, MA 02142 (stephanie.fradette@biogen.com).

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