Understanding Tofersen’s Impact on SOD1 ALS, Neurofilament Light: Timothy Miller, MD, PhD
The codirector of the ALS Center at Washington University School of Medicine in St. Louis discussed the long-term data of VALOR and its open-label extension assessing tofersen (Biogen) in SOD1 ALS. [WATCH TIME: 4 minutes]
WATCH TIME: 4 minutes
"The first [important finding] is that the levels of SOD1—the protein that we’re targeting—were lowered in the cerebrospinal fluid of the participants that were treated with the drug. This is good news. It shows the drug is doing exactly what it’s supposed to do."
There has been a palpable buzz within the amyotrophic lateral sclerosis (ALS) field as of recent, with the
In VALOR, 108 patients with SOD1-mutated ALS were randomized to tofersen 100 mg (n = 72) or placebo (n = 36) and treated for a 28-week treatment period. The combined analysis at week 52 of VALOR and its OLE was prespecified and intended to enable the comparison of early-start and delayed-start tofersen in the full intention-to-treat population. Another analysis has been planned when all participants have completed at least 3.5 years of follow-up, which has not yet been reached.2
At the
REFERENCES
1. Biogen announces FDA’s 3-month extension of review period for the new drug application for tofersen. News release. Biogen. October 17, 2022. Accessed October 17, 2022. https://www.globenewswire.com/news-release/2022/10/17/2535365/0/en/Biogen-Announces-FDA-s-3-Month-Extension-of-Review-Period-for-the-New-Drug-Application-for-Tofersen.html
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.
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