The European Medicines Agency (EMA) has recommended granting marketing authorisation in the European Union for a new therapy for the treatment of adult patients with amyotrophic lateral sclerosis (ALS), a rare and frequently fatal disease that causes muscle weakness and leads to paralysis. Qalsody (tofersen) is indicated for the treatment of adults with ALS who have a mutation in the SOD1 gene. There is currently only one treatment for ALS authorised in the EU (riluzole).
David Pozo - ELA EMA EN
David Pozo Pérez
Principal investigator of the Cellular and Molecular Neuroimmunology Laboratory at the Andalusian Centre for Molecular Biology and Regenerative Medicine (CABIMER)
The approval by the EMA (approximately one year after the FDA) of a drug based on ASOs (Antisense Oligonucleotide Therapy) technologies against a mostly genetic form of ALS, those patients with mutations in the SOD1 (superoxide dismutase 1) gene, which represent a very limited number of patients, is positive news. Among other things, it shows that the body of knowledge that we are acquiring at the molecular level is beginning to be transferred to patients.
It is true that the therapeutic effects are limited, as intervention on this mechanism, while evident at the molecular level, has less effect from a clinical point of view. These moderate effects translate into a slowing of motor degeneration and respiratory function, with a consequent improvement in patients' quality of life. It is not a cure for ALS, but it is certainly an important milestone; the disease has been described in the medical literature for just over 150 years. And, above all, it may represent one more tool to, in the near future, have combined strategies with different pharmacological approaches for an adequate clinical management of ALS.
