US biotechnology company Biogen Inc (Nasdaq:BIIB) revealed on Friday that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending a marketing authorisation under exceptional circumstances for QALSODY (tofersen) for the treatment of adults with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene.

If approved by the European Commission, QALSODY would be the first therapy approved in the European Union targeting this genetic cause of ALS.

The CHMP's recommendation is based on evidence including data from the Phase 3 VALOR study which showed a 60% reduction in plasma neurofilament light chain (NfL) levels in QALSODY-treated participants compared to placebo. Trends towards improvement in the physical abilities of participants who received QALSODY were seen compared to those who received placebo, as measured by the ALS Functional Ratings Scale-Revised (ALSFRS-R).

The recommendation for QALSODY under exceptional circumstances acknowledges the rarity of the disease. The Commission is expected to make a decision in the second quarter of 2024.

QALSODY, an antisense oligonucleotide (ASO), is licensed from Ionis Pharmaceuticals Inc (Nasdaq:IONS). It is also in Phase 3 trials for presymptomatic individuals with a SOD1 genetic mutation and biomarker evidence of disease activity.