Role of Tofersen Among Other Previously Approved ALS Therapies: Timothy Miller, MD, PhD


The codirector of the ALS Center at Washington University School of Medicine in St. Louis provided background on how tofersen would be used alongside other ALS medications if approved. [WATCH TIME: 3 minutes]

WATCH TIME: 3 minutes

"Many of us assume that a drug like tofersen for SOD1 ALS is targeting SOD1. That’s the cause of the disease. We’re very upstream, removing the gene product that most of us believe is causing ALS in people with this genetic disorder."

For about 90% of cases of amyotrophic lateral sclerosis (ALS), the person diagnosed is the only member of the family with the disease. The remaining 10% of cases, or familial ALS, are because more than 1 person in the family has had the disease, and thus, there may be a shared genetic mutation. Tofersen (Biogen), an antisense oligonucleotide, is currently in review with the FDA as a potential treatment for patients with SOD1 mutated-mediated ALS. If approved, it would become the first therapy to specifically focus on treating a form of familial ALS.

SOD1 ALS represents around 3% of all ALS cases worldwide. There are 3 therapies—riluzole (Rilutek), edaravone (Radicava; MT Pharma), and AMX0035 (Relyvrio; Amylyx Pharmacueticals)—that have been approved based on their ability to slow disease progression. Tofersen currently has a PDUFA date scheduled for April 25, 2023, and had its new drug application backed 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.

Principal investigator Timothy Miller, MD, PhD, codirector of the ALS Center at Washington University School of Medicine in St. Louis, recently sat down with NeurologyLive® to discuss the role of toferson in comparison with other previously approved therapies, as well as the advantages this new therapy may bring.

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.
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