Biogen has received the European Commission (EC) marketing authorisation for Qalsody (tofersen) to treat adults with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 gene (SOD1-ALS).
The EC has approved the drug under exceptional circumstances due to the rarity of the disease. However, the European Medicines Agency (EMA) recommended to maintain Qalsody’s designation as an orphan medicinal product.
Discovered by Ionis Pharmaceuticals, Qalsody is an antisense oligonucleotide (ASO) designed to target SOD1 mRNA to lower SOD1 protein production. Under a collaborative development and licensing deal, Biogen gained licence of QALSODY from Ionis Pharmaceuticals.
The US Food and Drug Administration (FDA) has already granted accelerated approval for the same indication in April last year.
The EC’s approval has been driven by the totality of evidence. This includes the targeted mechanism of action, biomarker, and clinical data.
The Phase 3 VALOR study involving 108 participants evaluated the ASO. Patients in the trial assigned in a 2:1 ratio to either receive Qalsody 100mg or placebo for 24 weeks.
The study’s primary efficacy endpoint was the change in the ALS Functional Ratings Scale-Revised total score from baseline to Week 28.
In the randomised, double-blind, and placebo-controlled trial, the results favoured tofersen numerically, but they did not reach statistical significance.
By Week 28, the mean plasma neurofilament light chain (NfL) levels, indicative of axonal injury and neurodegeneration, exhibited a 55% reduction in tofersen-treated participants compared to a 12% increase in the placebo group.
Biogen neuromuscular development unit head Stephanie Fradette said: “The European Commission’s approval of Qalsody is a testament to the unwavering dedication of the ALS community – people living with ALS and their loved ones, scientists, clinicians, and advocates – who have worked together over the past two decades to bring forward this important new treatment for the SOD1-ALS community.
“We are working with the medical community and local authorities to bring Qalsody to people living with SOD1-ALS across the region as quickly as possible.”
Apart from the ongoing open-label extension (OLE) of the VALOR study, the drug is being assessed in the Phase 3 ATLAS study.
This randomised, placebo-controlled ATLAS trial aims to assess whether tofersen can delay clinical onset when administered to presymptomatic individuals carrying a SOD1 genetic mutation and exhibiting biomarker evidence of disease activity.
