Special Episode: Tofersen Approved for SOD1 Amyotrophic Lateral Sclerosis

Angela Genge, MD, FRCPC

A special episode of the NeurologyLive^® Mind Moments^® podcast is now live! Scroll down to listen or click here to subscribe on your favorite streaming service.

The Mind Moments^® podcast features exclusive interviews with leaders in the field discussing the latest research and disease management strategies across the breadth of neurology, including epilepsy, multiple sclerosis (MS), Parkinson disease, dementia, sleep disorders, and more.

This episode, "Tofersen Approved for SOD1 Amyotrophic Lateral Sclerosis," features an exclusive interview with Angela Genge, MD, FRCPC, the director of the ALS Centre of Excellence for Research and Patient Care at McGill University, and an executive at the Clinical Research Unit at The Neuro (Montreal Neurological Institute-Hospital). Tofersen (Qalsody; Biogen/Ionis) was approved via the accelerated approval pathway, with supporting data from a phase 1 study of healthy volunteers, a phase 1/2 dose-ascending study, the pivotal phase 3 VALOR study (NCT02623699), and its open-label extension.^1,2

Click here to read more of NeurologyLive^®'s coverage of the tofersen approval.

EPISODE BREAKDOWN

• 0:30 – Tofersen (Qalsody; Biogen/Ionis) approved for SOD1-ALS
• 1:35 – Angela Genge, MD, FRCPC, on the approval
• 3:05 – The implications of targeting a root cause of ALS
• 5:00 – Data from the supporting VALOR study
• 5:55 – Genge on the context around the trial data
• 7:35 – Questions about access to tofersen amid accelerated approval

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REFERENCES
1. FDA approves treatment of amyotrophic lateral sclerosis associated with a mutation in the SOD1 gene. News release. FDA. April 25, 2023. Accessed April 25, 2023. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-treatment-amyotrophic-lateral-sclerosis-associated-mutation-sod1-gene
2. Miller TM, Cudkowicz ME, Genge A, et al. Trial of antisense oligonucleotide tofersen for SOD1 ALS. N Engl J Med. 2022;387:1099-1110. doi:10.1056/NEJMoa2204705.