Watch our oral testimony at the FDA Advisory Committee meeting on March 22, 2023.
We are grateful to the FDA Advisory Committee for reviewing the evidence, listening to the ALS community and voting to recommend accelerated approval of tofersen for treatment of SOD1-ALS.
Biogen presented compelling data suggesting that reductions in neurofilament levels predict slower progression of the disease. As we have followed the clinical trials, we have been encouraged by results that suggest an early start and extended use of tofersen may help stabilize muscle strength, respiratory function and quality of life. This is an enormously meaningful clinical benefit to people living with SOD1-ALS.
The committee heard powerful and heartfelt testimony from members of the ALS community, sharing the positive effect that tofersen has had on their daily lives. People treated with tofersen are living longer than their relatives and regaining abilities they had lost, like being able to walk upstairs unassisted.
The FDA’s accelerated approval pathway is conditional, meaning that people living with SOD1-ALS and clinicians will have access to tofersen while Biogen continues to collect clinical data on the drug’s effectiveness.
We believe that tofersen represents a significant advancement in the treatment of SOD1-ALS, and clinicians, researchers, advocates and people living with ALS agree. We are grateful to the people living with ALS who participated in these clinical trials and their caregivers, and to Biogen, the drug’s sponsor, for its commitment to this important work.
We urge the FDA to act on the committee’s recommendation for approval. The FDA is expected to provide their decision by April 25, 2023.