Biogen and Ionis Receive the US FDA’s Approval of Qalsody (tofersen) for the Treatment of Amyotrophic Lateral Sclerosis
Shots:
- 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
Click here to read the full press release
Neha is a Senior Editor at PharmaShots. She is passionate and very enthusiastic about recent updates and developments in the life sciences and pharma industry. She covers Biopharma, MedTech, and Digital health segments along with different reports at PharmaShots. She can be contacted at connect@pharmashots.com.
