Biogen’s Qalsody (tofersen) has been approved by the European Commission (EC) to treat amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene.
The decision follows a recent recommendation from the European Medicines Agency’s human medicines committee and makes Qalsody the first treatment approved in the EU to target a genetic cause of ALS.
Affecting an estimated 168,000 people globally, ALS is a rare and progressive neurodegenerative disease that results in the loss of motor neurons in the brain and the spinal cord that are responsible for controlling voluntary muscle movement.
Multiple genes have been implicated with ALS and mutations in the SOD1 gene are responsible for approximately 2% of all cases of the disease.
Qalsody, which has maintained its designation as an orphan medicinal product, is an antisense oligonucleotide that is designed to bind to SOD1 mRNA to reduce SOD1 protein production.
The drug has been granted marketing authorisation under exceptional circumstances, which is recommended when a treatment’s benefit/risk assessment is determined to be positive, but it is unlikely that comprehensive data can be obtained under normal conditions of use due to the rarity of the disease.
The EC’s decision is based on a totality of evidence, Biogen said, including data from the late-stage VALOR study.
Results from VALOR showed that patients treated with Qalsody experienced significant reductions in plasma neurofilament light chain, a marker of neurodegeneration, compared to the placebo cohort.
Trends towards improvement in the physical abilities of Qalsody-treated patients were also seen compared to those who received placebo, as measured by the ALS Functional Ratings Scale-Revised at week 28.
Stephanie Fradette, head of the neuromuscular development unit at Biogen, described the approval as a “testament to the unwavering dedication” of the ALS community, adding that the company is now “working with the medical community and local authorities to bring Qalsody to people living with SOD1-ALS across the region as quickly as possible”.
Qalsody, which Biogen has licensed from Ionis Pharmaceuticals, was granted accelerated approval by the US Food and Drug Administration in April last year for the same patient population.