Biogen and Ionis’ ALS Treatment Qalsody Receives European Commission Approval

1. European Commission Approval: The European Commission granted marketing authorization to Biogen and Ionis' Qalsody (tofersen) for treating adults with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 gene (SOD1-ALS).
2. First Genetic ALS Treatment: Qalsody is the first therapy approved in the European Union to target a genetic cause of ALS, also known as motor neuron disease (MND).
3. Rare Disease: SOD1-ALS is an ultra-rare genetic form of ALS, estimated to affect less than 1,000 people in Europe.
4. Exceptional Circumstances: The marketing authorization was granted under exceptional circumstances due to the rarity of the disease, making it unlikely to obtain comprehensive data under normal conditions of use.
5. Neurofilament Biomarker: Qalsody has helped advance neurofilament as a tool to optimize clinical trial design in ALS, offering the potential to expedite further breakthroughs in the field.
6. Phase 3 VALOR Study: The approval is based on the totality of evidence, including the targeted mechanism of action, biomarker, and clinical data from the randomized, double-blind, placebo-controlled Phase 3 VALOR study.
7. Biogen's Commitment: Qalsody is Biogen's third rare disease therapy to be approved in the EU, demonstrating the company's commitment to addressing diseases with a high unmet need.