Pharma Focus Asia

Biogen Develops QALSODY®, the First Therapy to Treat a Rare, Genetic Form of ALS

Biogen has developed QALSODY® (tofersen), a groundbreaking therapy for adults with amyotrophic lateral sclerosis (ALS) linked to a mutation in the superoxide dismutase 1 gene (SOD1-ALS).

QALSODY® (tofersen) operates as an antisense oligonucleotide (ASO), specifically designed to bind to SOD1 mRNA, thereby reducing the production of SOD1 protein. 

The approval of QALSODY is grounded in a comprehensive evaluation, including its targeted mechanism of action, biomarker data, and clinical evidence. In the Phase 3 VALOR study, a randomised, double-blind, placebo-controlled trial involving 108 participants, patients were assigned in a 2:1 ratio to receive either QALSODY 100 mg or placebo for 24 weeks. 

ALS is a rare, progressively debilitating disease characterised by the loss of motor neurons in the brain and spinal cord, leading to a decline in voluntary muscle movement. Individuals with ALS endure muscle weakness and atrophy, gradually losing their ability to move, speak, eat, and ultimately breathe.

With the approval of QALSODY in Europe, individuals with SOD1-ALS will now have access to the first treatment targeting a genetic root cause of ALS. This signifies a significant milestone for the ALS community, offering hope that ALS is indeed a manageable condition.

The U.S. Food and Drug Administration has granted accelerated approval for QALSODY to address ALS in adults carrying a mutation in the superoxide dismutase 1 (SOD1) gene. The accelerated approval was granted due to the observed decline in plasma neurofilament light chain (NfL) levels in patients who received treatment with QALSODY.

 

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