Oligonucleotide ALS therapy approved in EU

The European Commission (EC) has granted Biogen its third rare disease treatment approval in the European Union.


The first treatment approved in the EU to target a genetic cause of amyotrophic lateral sclerosis (ALS), has been authorised by the European Commission (EC). QALSODY ® (tofersen) is indicated for adults with ALS associated with a mutation in the superoxide dismutase 1 gene (SOD1-ALS). The treatment is an antisense oligonucleotide (ASO) designed to bind to SOD1 mRNA to reduce SOD1 protein production, Biogen shared.

This authorisation is subject to approval under exceptional circumstances and orphan designation of QALSODY. Biogen stated that it licensed the treatment from Ionis Pharmaceuticals, Inc.

[The EC’s approval is] a major milestone… showing that ALS is a treatable disease”

Notably, with QALSODY, by helping to advance neurofilament “as a tool to optimise clinical trial design in ALS”, this opens potential opportunity for future breakthroughs in the field, Biogen noted.

The EC’s approval is “a major milestone… showing that ALS is a treatable disease,” stated Evy Reviers, Chairwoman of the European Organisation for Professionals and People living with ALS (EUpALS).

Clinical evaluation of QALSODY (tofersen)

Findings from the Phase III VALOR study showed that at Week 28, mean plasma neurofilament light chain (NfL), a marker of axonal injury and neurodegeneration, decreased by 55 percent in individuals given tofersen. This is compared to a 12 percent increase in patients given placebo treatment.

“The European Academy of Neurology has confirmed new treatment guidelines for ALS that recognise QALSODY should be offered as first-line treatment for patients with SOD1-ALS,” explained Dr Philip Van Damme, PhD, Professor of Neurology and Director of the Neuromuscular Reference Center at the University Hospital Leuven in Belgium.

Further confirmatory clinical evidence may be required for continued approval of QALSODY for this indication, according to Biogen.

QALSODY is also being evaluated in the Phase III ATLAS study. This trial is investigating evaluate whether the therapy can delay clinical onset when initiated in presymptomatic individuals with a SOD1 genetic mutation.

The European Medicines Agency (EMA) accepted the marketing authorisation application (MAA) for tofersen, to treat SOD1 ALS in December 2022.