EU medicines regulator backs Biogen ALS drug

The CHMP’s recommendation for QALSODY is based on the totality of evidence, including the targeted mechanism of action, biomarker and clinical data. In the 28-week Phase 3 VALOR study, reductions of 60% in plasma neurofilament light chain (NfL) were observed in participants who received QALSODY compared to the placebo group, suggesting reduced neuronal injury. 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 CHMP’s recommendation in support of QALSODY approval provides new hope for the ALS community in Europe,” said Philip Van Damme, M.D., Ph.D., professor of neurology and director of the Neuromuscular Reference Center at the University Hospital Leuven in Belgium. “This is a significant milestone for the entire ALS community - for the first time we have a treatment that led to sustained reductions in neurofilament, a marker of axonal injury and neurodegeneration. The QALSODY development program has provided critical learnings on clinical trial design and the use of biomarkers that is advancing the entire field.”

A marketing authorization under exceptional circumstances is recommended when the benefit/risk assessment is determined to be positive but due to the rarity of the disease, it is unlikely that comprehensive data can be obtained under normal conditions of use. The CHMP’s recommendation for QALSODY will now be reviewed by the EC for a decision on a marketing authorization in the European Union, with a decision expected in the second quarter of 2024.