FDA Adcomm Says Benefit-Risk Profile Favor Biogen's Tofersen For Inherited Neuro Disorder

  • The FDA's Peripheral and Central Nervous System Drugs Advisory Committee voted 9 yes - 0 no, indicating sufficient evidence showing a reduction in plasma neurofilament light chain (NfL) concentration in tofersen-treated patients is reasonably likely to predict the clinical benefit of tofersen for SOD1-ALS patients.
  • Biogen Inc BIIB licensed tofersen from Ionis Pharmaceuticals Inc IONS under a collaborative development and license agreement.
  • Neurofilament light chain (NfL) is a biomarker of neuronal damage.
  • Related: Despite Failed Phase 3 Study, FDA Staff Says Biogen's ALS Drug May Have Clinical Benefit.
  • The panel voted 5 No - 3 Yes (1 Absent) that the late-stage data for the drug, tofersen, did not show the effects on relevant biomarkers (i.e., changes in plasma NfL concentration and/or reductions in accumulated toxic protein levels known as superoxide dismutase 1 (SOD1).
  • Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, results in weakened muscles and causes nerve cells in the brain and spinal cord to break down, affecting physical function and leading to severe disability and death.
  • The committee reached a consensus that the benefit-risk profile was favorable based on reviewing the totality of data for tofersen in people with SOD1-ALS.
  • The FDA is continuing its review of tofersen with a Prescription Drug User Fee Act action date of April 25, 2023.
  • Price Action: BIIB shares closed 1.88% higher at $266.15 during after-hours trading on Wednesday.
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