The European Commission (EC) has granted marketing authorisation for Biogen's QALSODY (tofersen) an antisense oligonucleotide, to treat adults with amyotrophic lateral sclerosis (ALS) linked to a mutation in the superoxide dismutase 1 gene (SOD1-ALS).
This is the first approval in the European Union for a therapy targeting a genetic cause of ALS, a motor neuron disease.
QALSODY received the authorisation under exceptional circumstances but due to the rarity of the disease, it is challenging to gather comprehensive data.
The European Medicines Agency also recommended maintaining QALSODY's orphan medicinal product designation.
The latest approval is based on the totality of evidence, including the mechanism of action, biomarkers and clinical findings from the Phase III VALOR study.
108 patients were randomised to receive either QALSODY 100mg or a placebo for 24 weeks.
The variation in the ALS functional ratings scale-revised total score at week 28 from baseline was the primary efficacy endpoint.
While the results numerically favoured tofersen, they did not reach statistical significance. However, a significant reduction in plasma neurofilament light chain, a marker of neurodegeneration, was observed in the tofersen-treated group.
Common adverse reactions observed in the trial included pain, fatigue, muscle and joint pain, fever and changes in the cerebrospinal fluid.
QALSODY is approved in the US, and Biogen is in discussions with regulatory authorities in other regions.
The therapy was discovered by Ionis Pharmaceuticals and licensed to Biogen under a collaborative deal.
Biogen neuromuscular development unit head Stephanie Fradette stated: “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.”
Biogen recently entered a definitive agreement for the acquisition of clinical-stage biotechnology company Human Immunology Biosciences in a $1.8bn deal.
