European Commission approves Qalsody(tofersen) to treat adults with amyotrophic lateral sclerosis at adults with amyotrophic lateral sclerosis (ALS) linked to a mutation in the superoxide dismutase 1 gene (SOD1-ALS)
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.
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