Welcome to this special episode of the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. For major FDA decisions in the field of neurology, we release short special episodes to offer a snapshot of the news, including the main takeaways for the clinical community, as well as highlights of the efficacy and safety profile of the agent in question.

In this episode, we're covering the recent approval of tofersen (Qalsody; Biogen/Ionis) in SOD1 mutation-mediated amyotrophic lateral sclerosis (ALS). Offering commentary is 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 is an antisense oligonucleotide, and its new drug application (NDA) was supported by 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.

For more of NeurologyLive®'s coverage of tofersen's approval, head here: FDA Approves Tofersen as First SOD1-ALS Treatment

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

Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

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.