![]() Of these participants, 95 enrolled in the ongoing OLE. In total, 108 participants were randomized in VALOR (n=72 to tofersen 100 mg and n=36 to placebo). VALOR was a six-month Phase 3, randomized, double-blind, placebo-controlled study to evaluate the effects of tofersen 100 mg in adults with ALS associated with a SOD1 mutation. “The lowering of neurofilament, a marker of axonal injury and neurodegeneration along with the clinical data, highlights the potential of tofersen.” We are now seeing in the data what we suspected about tofersen for a long time – that it has the potential to make a clinical difference for people living with SOD1-ALS,” said Merit Cudkowicz, M.D., co-principal investigator of the VALOR trial and co-founder of the Northeast ALS Consortium, Director of the Healey & AMG Center for ALS and Chair of Neurology at Massachusetts General Hospital and the Julieanne Dorn Professor of Neurology at Harvard Medical School. To have data like these published in NEJM gives us energy and hope. “The ALS community has been actively pursuing new medicines for decades. ![]() The application was given a Prescription Drug User Fee Act action date of January 25, 2023. “We are grateful to the dedication from participants, their families, and the sites for taking part in this important study.”ĭata from the combined analysis were previously presented at the European Network to Cure ALS (ENCALS) annual meeting and included within Biogen’s New Drug Application for tofersen that was recently accepted for priority review by the U.S. And third, there is a meaningful clinical benefit when looking at the later time points in the open label extension,” said Timothy Miller, M.D., Ph.D., principal investigator of VALOR and ALS Center co-Director at Washington University School of Medicine, St. “I see three key take home points from these data. First, tofersen clearly leads to lowering of SOD1 protein, as would be expected. Second there is substantial lowering of neurofilament levels, which I interpret as potentially slowing the underlying disease process. ![]() There is currently no treatment targeted for SOD1-ALS. (Nasdaq: BIIB) today announced that The New England Journal of Medicine (NEJM) has published detailed results from the Phase 3 VALOR study and the combined analysis of VALOR and its open label extension (OLE) study evaluating tofersen for the treatment of superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). ![]()
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