The US FDA has granted accelerated approval to Qalsody (100mg/15mL) for adults with ALS who have a mutation in the SOD1 gene. The indication was approved based on a reduction in plasma NfL
The approval was based on 28wk. P-III study (VALOR) evaluating tofersen vs PBO in a ratio (2:1) in 108 adults aged 23-78yrs. In the primary analysis population, less decline from baseline as measured by ALSFRS-R & the results were not statistically significant
In the overall ITT population, 55% reduction in plasma NfL vs 12% increase in PBO. Levels of CSF SOD1 protein, an indirect measure of target engagement was reduced by 35% vs 2%. Findings from an interim analysis at 52wks. showed similar reductions in NfL in patients previously receiving PBO who initiated tofersen in OLE & those who received tofersen in 28wk. study
Ref: GlobeNewswire | Image: Biogen
Related News:- Eisai and Biogen Report the US FDA’s Acceptance of sBLA and Granted Priority Review for Leqembi (lecanemab-irmb) to Treat Alzheimer’s Disease
