Spain Approves Groundbreaking Genetic ALS Treatment Tofersen for Public Use

The Interministerial Commission on Drug Prices (CIPM) in Spain has approved the funding of Tofersén, an innovative treatment for amyotrophic lateral sclerosis (ALS), marketed under the name Qalsody. With this decision, according to the Ministry of Health, Spain is among the first European countries to incorporate this therapy into the public health system.

Pioneering Move Places Spain at the Forefront of Personalized ALS Care with EU’s First Gene-Targeted Therapy

Tofersen is the first drug approved in the European Union that directly targets a known genetic cause of ALS: mutations in the SOD1 gene, responsible for a hereditary form of the disease that affects approximately 2% of diagnosed patients. This genetic alteration causes the abnormal accumulation of a toxic protein that damages motor neurons, cells essential for controlling muscle movement.

The drug acts through a gene silencing mechanism that reduces the production of this protein, with the goal of preserving neuronal function and slowing disease progression. Tofersen represents one of the first therapies targeting a specific genetic cause of ALS, marking a milestone in the personalized approach to this neurodegenerative disease.

The drug’s use requires identification of the mutated SOD1 protein through a test available at most ALS referral centers, which are present in most autonomous communities in Spain.

Spain Leads the Way in ALS Innovation with Early Adoption of Game-Changing Genetic Treatment

The medicine was authorized by the European Medicines Agency (EMA) in May 2024 under exceptional circumstances, given that it is a rare disease and the available clinical data are limited. Tofersen also has orphan medicinal product designation, which recognizes its therapeutic value in conditions of high unmet medical need. The European Medicines Agency will review any new information about the medicine that may become available annually, and its summary of product characteristics will be updated as necessary.

On May 19th, 2025, the Spanish Agency for Medicines and Medical Devices (AEMPS) published its therapeutic positioning report for this drug, which considers tofersen as a therapeutic option for the treatment of adults with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene that has demonstrated an effect on biomarkers related to its mechanism of action.

Formal confirmation of clinical benefit will require additional mature efficacy and safety data and annual re-evaluation of the evidence as it develops. Spain positions at the forefront.

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First published in diariofarma . A third-party contributor translated and adapted the article from the original. In case of discrepancy, the original will prevail.

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