Biogen announced that the European Commission (EC) has granted marketing authorisation under exceptional circumstances and maintained orphan designation for QALSODY (tofersen) for the treatment of adults with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 gene (SOD1-ALS). QALSODY is the first treatment approved in the European Union to target a genetic cause of ALS, also known as motor neuron disease (MND).
The marketing authorisation for QALSODY is granted under exceptional circumstances, which is recommended when the benefit/risk assessment of a treatment is determined to be positive but due to the rarity of the disease, it is unlikely that comprehensive data can be obtained under normal conditions of use. The European Medicines Agency (EMA) recommended QALSODY’s designation as an orphan medicinal product be maintained.
The approval of QALSODY is based on the totality of evidence, including the targeted mechanism of action, biomarker, and clinical data. In the randomised, double-blind, placebo-controlled Phase 3 VALOR study (n=108), patients were randomised 2:1 to receive treatment with either QALSODY 100 mg (n=72) or placebo (n=36) for 24 weeks. The primary efficacy endpoint was the change from baseline to Week 28 in the ALS functional ratings scale-revised total score.
The results numerically favoured tofersen, but were not statistically significant (ITT population: tofersen-placebo adjusted mean difference [95 per cent CI]: 1.4 [-1.3, 4.1]). At Week 28, mean plasma neurofilament light chain (NfL), a marker of axonal injury and neurodegeneration, was reduced by 55 per cent (geometric mean ratio to baseline) in the tofersen-treated participants (ITT), compared to a 12 per cent increase with placebo (difference in geometric mean ratios for tofersen to placebo: 60 per cent (95 per cent CI: 51 per cent, 67 per cent)).
Very common adverse reactions (may affect more than 1 in 10 people) reported in QALSODY-treated participants were pain (back pain, pain in arms or legs), feeling tired, muscle and joint pain, fever, and an increase in protein and/or white blood cell count occurring in the fluid that surrounds the brain and spinal cord.
Through the Biogen early access program, about 330 people with SOD1-ALS have received QALSODY across 18 EU countries. QALSODY is also approved for use in the United States and Biogen is engaging with regulatory authorities in other regions.
About QALSODY (tofersen)
QALSODY (tofersen) is an antisense oligonucleotide (ASO) designed to bind to SOD1 mRNA to reduce SOD1 protein production. The US FDA granted accelerated approval for QALSODY to treat amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. This indication is approved under accelerated approval based on reduction in plasma neurofilament light chain (NfL) observed in patients treated with QALSODY. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s). The European Commission granted marketing authorisation under exceptional circumstances and orphan designation for QALSODY.
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