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Biogen Gains European Commission Approval for QALSODY (tofersen), a Novel Therapy for a Rare Genetic Form of ALS | EMA Drug Approvals | 2024 | iPharmaCenter

Biogen has announced that the European Commission (EC) has granted marketing authorization for QALSODY (tofersen), marking the first therapy approved in the European Union to address a genetic cause of amyotrophic lateral sclerosis (ALS), specifically the form linked to mutations in the superoxide dismutase 1 (SOD1) gene. This rare and fatal genetic type of ALS, affecting fewer than 1,000 individuals in Europe, is now treatable with QALSODY.

Biogen stated that QALSODY's approval under exceptional circumstances underscores the positive benefit-risk assessment of the treatment, even though the condition's rarity makes it challenging to gather comprehensive data. The European Medicines Agency (EMA) has also upheld its designation as an orphan medicinal product.

The authorization is based on a thorough review of the therapy's mechanism, biomarkers, and clinical trial data. In the Phase 3 VALOR study, which involved 108 patients, participants were assigned in a 2:1 ratio to receive either QALSODY or a placebo over 24 weeks. The primary measure of effectiveness was the change in the ALS Functional Rating Scale-Revised score from the baseline to week 28. Although the results numerically favored QALSODY, they were not statistically significant. At week 28, there was a notable 55% reduction in the plasma neurofilament light chain (NfL), a marker of nerve cell damage, in those treated with QALSODY compared to a 12% increase in the placebo group.

Common side effects reported in participants treated with QALSODY included pain (in the back, arms, or legs), fatigue, muscle and joint pain, fever, and increased levels of protein and white blood cells in the cerebrospinal fluid.

Biogen’s early access program has provided QALSODY to approximately 330 individuals with SOD1-ALS in 18 European countries. QALSODY is also approved in the United States, and Biogen is actively engaging with regulatory bodies in other regions to expand availability.

QALSODY (tofersen) is an antisense oligonucleotide (ASO) specifically designed to bind to SOD1 mRNA and reduce the production of the SOD1 protein. In the U.S., it received accelerated approval from the FDA for treating ALS in adults with SOD1 gene mutations. This approval was based on reductions in plasma NfL observed in treated patients, with continued approval contingent upon verification of clinical benefits in further trials. The European Commission also granted marketing authorization under exceptional circumstances and orphan designation for QALSODY.

Biogen obtained the rights to QALSODY through a licensing agreement with Ionis Pharmaceuticals, Inc., the original developer of the treatment.

Ongoing studies include the open-label extension of the Phase 3 VALOR study and the Phase 3 ATLAS study, which aims to determine if QALSODY can delay disease onset in presymptomatic individuals with SOD1 mutations and elevated plasma NfL levels.

Amyotrophic lateral sclerosis (ALS) is a rare, progressive neurodegenerative disease that causes the loss of motor neurons responsible for voluntary muscle movements, leading to muscle weakness, atrophy, and eventual loss of movement, speech, and respiratory functions. The average life expectancy after symptom onset is three to five years.

Several genetic mutations have been linked to ALS, and genetic testing can identify these mutations even in patients without a family history of the disease. SOD1 gene mutations account for about 2% of ALS cases worldwide. These mutations result in the production of a toxic, misfolded SOD1 protein, leading to motor neuron degeneration, progressive muscle weakness, and death.


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