The FDA has approved Biogen's ALS drug, Tofersen, an antisense oligo targeting SOD1. I wrote about this back in September in my Clinical Pipeline column as part of a shift towards approving drugs based on biomarkers without clear signs of clinical efficacy*.
https://www.nature.com/articles/d41591-022-00098-w
*You can have a look at the trial data in NEJM, in a study that concluded that "In persons with SOD1 ALS, tofersen reduced concentrations of SOD1 in CSF and of neurofilament light chains in plasma over 28 weeks but did not improve clinical end points and was associated with adverse events."
#NatureMedicine #amyotrophiclateralsclerosis #drugdevelopment #pharma
And a more recent piece by Asher Mullard for Nature
"A thumbs-up for tofersen from the US Food and Drug Administration would reinforce the agency’s shifting approach to neurological drugs."
Here's a link to the FDA's decision:
"FDA approved Qalsody (tofersen) to treat patients with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS)."
https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-treatment-amyotrophic-lateral-sclerosis-associated-mutation-sod1-gene