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FDA Accepts Biogen’s New Drug Application and Grants Priority Review of Tofersen for a Rare, Genetic Form of ALS
SOD1-ALS is a rare genetic form of ALS that affects approximately 330 people in the U.S.,1 it is progressive, leads to the loss of everyday functions and is
About this update from Biogen Inc.
[{"type":"text","content":"SOD1-ALS is a rare genetic form of ALS that affects approximately 330 people in the U.S.,1 it is progressive, leads to the loss of everyday functions and is uniformly fatalIf approved, tofersen would be the first treatment to target a genetic cause of ALS12-month data included in the filing show that earlier initiation of tofersen slowed decline across measures of clinical and respiratory function, strength, and quality of life CAMBRIDGE, Mass., July 26, 2022 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) today announced that the U.S. Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for tofersen, an investigational drug for superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). The application has been granted priority review and given a Prescription Drug User Fee Act action date of January 25, 2023. The FDA has noted that it is currently planning to hold an Advisory Committee meeting for this application, on a yet-to-be determined date. The average life expectancy for people with ALS is three to five years from time of symptom onset. There is currently no treatment targeted for SOD1-ALS.2 “The available data show that tofersen has the potential to make a meaningful difference for people with SOD1-ALS,” said Priya Singhal, M.D., M.P.H., Head of Global Safety and Regulatory Sciences and Interim Head of R&D at Biogen. “Pursuing the FDA’s accelerated approval pathway offers the potential to make tofersen available to people living with this fatal, neurodegenerative disease as quickly as possible. If approved, tofersen will be the first treatment to target a genetic cause of ALS and we hope this will pave the way for further advances in this relentless disease.” Biogen is seeking approval of tofersen under the FDA’s accelerated approval pathway, based on the use of neurofilament as a surrogate biomarker that is reasonably likely to predict clinical benefit. Neurofilaments are normal proteins found in healthy neurons, that are increased in blood and cerebrospinal fluid when damage has been done to neurons or their axons and are a marker of neurodegeneration. In ALS, higher levels of neurofilaments have been found to predict more rapid decline in clinical function and shortened survival.3 Tofersen study results suggest reductions in neurofilament preceded and predicted slowing of decline in meas...