Biogen Gains EU Approval for QALSODY® ALS Therapy

Biogen Inc. has announced that the European Commission (EC) has granted marketing authorization for QALSODY® (tofersen), a new treatment specifically designed for adults with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 gene (SOD1-ALS). This approval marks the first time a treatment targeting a genetic cause of ALS has been approved in the European Union. SOD1-ALS is a particularly aggressive and rare form of ALS, affecting fewer than 1,000 individuals in Europe.

QALSODY's approval under exceptional circumstances reflects its potential benefits despite the limited data available due to the rarity of SOD1-ALS. The European Medicines Agency (EMA) has maintained QALSODY's designation as an orphan medicinal product. The authorization is based on comprehensive evidence, including a Phase 3 VALOR study, which although showed numerical favor towards tofersen, did not achieve statistical significance in its primary endpoint. However, a significant reduction in neurofilament light chain (NfL), a marker of neurodegeneration, was observed in tofersen-treated patients compared to those on placebo.

The announcement of QALSODY’s approval has been met with optimism by the ALS community. Stephanie Fradette, head of the Neuromuscular Development Unit at Biogen, emphasized the collaborative efforts over the past twenty years by patients, scientists, and advocates in bringing this treatment to fruition. Similarly, Philip Van Damme, a neurology professor at the University Hospital Leuven, highlighted the treatment's potential to change the landscape of SOD1-ALS therapy and offer new hope to patients and their families.

QALSODY’s development has also advanced the use of neurofilament as a biomarker to fine-tune clinical trial designs in ALS, potentially accelerating future treatment breakthroughs. Biogen has been instrumental in this progress, demonstrating a commitment to addressing diseases with high unmet needs. The company has also ensured early access to QALSODY for around 330 patients across 18 EU countries through its early access program.

Besides the European Union, QALSODY is approved in the United States, and Biogen is actively engaging with regulatory authorities in additional regions to broaden access. The treatment’s mechanism involves binding to SOD1 mRNA to reduce the production of the SOD1 protein, thereby aiming to mitigate the toxic effects of the misfolded protein that leads to motor neuron degeneration in SOD1-ALS patients.

ALS is a fatal neurodegenerative condition characterized by the loss of motor neurons responsible for voluntary muscle movements, leading to progressive muscle weakness and eventual death. The average life expectancy of ALS patients is three to five years post-diagnosis. Genetic mutations, including those in the SOD1 gene, are implicated in a subset of ALS cases.

For over a decade, Biogen has been dedicated to ALS research, focusing on both genetic and non-genetic forms of the disease. Their efforts have included the evaluation of genetically validated targets and employing sensitive clinical endpoints to enhance the likelihood of successful treatment outcomes. Alongside QALSODY, Biogen continues to explore other therapeutic avenues, including addressing TDP43 pathology present in the majority of ALS cases.

Founded in 1978, Biogen remains at the forefront of biotechnology, striving to develop innovative treatments to transform patients' lives while delivering value to shareholders and the broader community. The company’s approach balances scientific risk with investment returns, aiming for long-term growth and success in the biotechnology sector.