Biogen announced that the final results of its Phase 3 VALOR study and the subsequent open‑label extension for QALSODY (tofersen) in patients with superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS) have been published in the Journal of the American Medical Association Neurology on December 12, 2025.
The 108‑patient study followed participants for a median of 4.9 years. Early initiation of QALSODY was associated with a numerically slower decline in clinical function, breathing, and strength, and a reduced risk of death or permanent ventilation. Neurofilament light chain levels were also lowered, confirming the drug’s disease‑modifying effect in this genetically defined ALS subset.
QALSODY received accelerated approval from the U.S. Food and Drug Administration in January 2023 for the treatment of SOD1‑ALS, based on its ability to reduce neurofilament light chain. The JAMA Neurology publication provides the long‑term confirmatory data that Biogen and regulators will use to support continued use and to explore potential label expansions.
These data reinforce Biogen’s strategy to broaden its ALS portfolio beyond its broader‑population SMA therapy, SPINRAZA. By adding a high‑margin product that addresses a rare, genetically defined subset of ALS, Biogen positions itself to capture a growing niche market that is expected to expand as gene‑editing and antisense therapies mature.
SOD1‑ALS accounts for roughly 2 % of all ALS cases, a population that has historically had limited treatment options. The confirmatory data demonstrate that tofersen can alter disease trajectory in this group, providing a meaningful therapeutic option and strengthening Biogen’s reputation as a leader in rare‑disease neurology.
The publication of these final results signals a key milestone for Biogen’s ALS program and supports the company’s broader focus on high‑margin, high‑impact therapies in rare neurological disorders.
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