The Ministry of Health, through the Interministerial Commission of Price of Medicines (CIPM), has given the green light to the financing of Tofersan innovative treatment against amyotrophic lateral sclerosis (ELA), marketed with the name Glassed®. This decision places Spain among the pioneering countries in Europe to incorporate this therapy into the public health system.
Tofersén is the first authorized medication in the European Union that acts directly on a known genetic cause of the ELA: Mutations in the SOD1 gene, responsible in an inherited way of the disease that affects approximately 2% of patients. This mutation causes the abnormal accumulation of a toxic protein that damages motor neurons, fundamental for the control of muscle movements.
The drug uses a gene silent strategy to reduce the production of this protein, with the aim of protecting neuronal function and curbing the evolution of the disease. It is one of the First therapies specifically aimed at a genetic cause of the ELAwhich means a significant advance towards a more personalized approach in the treatment of this neurodegenerative disease.
For use, it is necessary to identify the Mutation in the Sod1 protein through a diagnostic test that is already available in many of the ELA reference centers distributed by various autonomous communities.
The European Medicines Agency (EMA) approved the use of Tofersén in May 2024 under “exceptional circumstances”, since it is a rare pathology and clinical evidence is still limited. In addition, the medicine has been designated as an orphan, a category that recognizes its therapeutic potential in diseases with high non -covered medical need. EMA will review the new clinical information available on the drug every year and update its technical file if necessary.
On May 19, 2025, the Spanish Agency for Medicines and Health Products (AEMPS) published the therapeutic positioning report of Tofersén, in which it is considered a therapeutic alternative for adults with ELA linked to a mutation in the overexide gene Dysmutase 1 (SOD1). The medicine has shown effect on biomarkers related to its mechanism of action. However, the definitive validation of its clinical benefit will depend on the collection of more solid data on its effectiveness and safety, as well as a continuous annual evaluation of the new evidence.
Tofersén will be incorporated into the pharmaceutical benefit of the National Health System, which represents a relevant innovation in a therapeutic field that had not registered pharmacological advances since the 1990s. It is not a cure, but a directed therapy that marks a before and after in the personalized approach of this disease, since it is one of the first times that it is possible to act directly on a known genetic cause of the ELA.
Source: www.webconsultas.com