Biogen’s QALSODY® (tofersen) Approved by European Commission for Rare Genetic ALS Treatment

The European Commission (EC) has granted marketing authorization, maintaining orphan designation, for QALSODY® (tofersen) to treat amyotrophic lateral sclerosis (ALS) in adults associated with a mutation in the superoxide dismutase 1 gene (SOD1-ALS). QALSODY marks the first approved therapy in the European Union targeting a genetic cause of ALS, also known as motor neuron disease (MND).

Stephanie Fradette, Pharm.D., Head of the Neuromuscular Development Unit at Biogen, expressed gratitude to the ALS community for their two-decade-long collaboration in bringing this treatment forward. Fradette emphasized Biogen’s commitment to swiftly making QALSODY accessible to individuals with SOD1-ALS across the region.

QALSODY’s approval under exceptional circumstances reflects a positive benefit/risk assessment despite the rarity of the disease, as recommended by the European Medicines Agency (EMA). Notably, the approval is based on comprehensive evidence, including targeted mechanism of action, biomarker, and clinical data.

Philip Van Damme, M.D., Ph.D., Professor of Neurology and Director of the Neuromuscular Reference Center at the University Hospital Leuven in Belgium, hailed QALSODY’s approval as a paradigm shift in SOD1-ALS treatment. The European Academy of Neurology has updated ALS treatment guidelines to prioritize QALSODY as first-line therapy for SOD1-ALS patients.

The pivotal Phase 3 VALOR study, involving 108 participants, demonstrated favorable results though not statistically significant in the primary efficacy endpoint. However, QALSODY treatment notably reduced mean plasma neurofilament light chain (NfL), a marker of neurodegeneration, compared to placebo.

Evy Reviers, Chairwoman of the European Organisation for Professionals and People living with ALS (EUpALS), expressed excitement about QALSODY’s approval, marking a significant advancement in ALS treatment and underscoring ALS as a treatable disease.

Biogen reaffirmed its dedication to facilitating QALSODY access for eligible European patients, citing the successful early access program benefiting around 330 individuals across 18 EU countries. QALSODY is also approved in the United States, with Biogen actively engaging regulatory authorities in other regions.

QALSODY® (tofersen) is an antisense oligonucleotide (ASO) designed to reduce SOD1 protein production. Its accelerated approval by the U.S. FDA and marketing authorization by the European Commission highlight its importance in treating ALS.

The article further discusses ongoing studies like the Phase 3 ATLAS trial and provides insights into ALS and SOD1-ALS, emphasizing Biogen’s persistent commitment to ALS research despite past setbacks.

Biogen’s dedication to ALS research spans over a decade, focusing on genetically validated targets and appropriate modalities to address various forms of the disease, including TDP43 pathology, a hallmark of ALS cases.

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