The U.S. Food and Drug Administration (FDA) has accepted Biogen’s BIIB New Drug Application (NDA) for its lead asset Tofersen for superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS).
The regulatory agency as granted priority review and assigned PDUFA action date of January 25, 2023.
The New Drug Application (NDA) is supported by the results from the Phase 1 study in healthy volunteers, a Phase 1/2 study evaluating ascending dose levels, the Phase 3 VALOR study, and the open label extension (OLE) study.
The agency is currently planning to hold an Advisory Committee meeting for this new drug application.
Priya Singhal, Head of Global Safety and Regulatory Sciences and Interim Head of R&D, Said, "The available data show that tofersen has the potential to make a meaningful difference for people with SOD1-ALS. 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.”
The company 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.
Price Action : Biogen shares are trading up at $208 on Tuesday during pre-market session.
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