StoryRegulatory
EU approves Qalsody for SOD1-ALS under exceptional circumstances
The European Commission authorised tofersen for adults with SOD1-ALS, the first EU treatment aimed at a genetic cause of ALS. The approval was granted under exceptional circumstances, used when full data are hard to collect in a rare disease, and Biogen may need to provide more confirmatory evidence. The European Academy of Neurology now recommends it as first-line treatment for SOD1-ALS.
- Basis: VALOR, with plasma neurofilament down 55% vs up 12% on placebo at week 28.
- Orphan designation; Biogen's third rare-disease approval in the EU.