French authorities have denied early access to the drug Qalsody (Tofersen) for patients with amyotrophic lateral sclerosis (ALS) with a SOD1 gene mutation, despite its recent approval in the United States and Europe.
This decision provokes the anger of patients and associations, in particular the Association for Research on ALS (ARSLA), which cites unequal treatment. ALS, or Charcot disease, is a neurodegenerative disease leading to progressive paralysis. Qalsody, specifically targeting the SOD1 mutation, is the only treatment available for this rare form of ALS. In France, several dozen patients benefited from compassionate access to the drug.
The High Authority of Health (HAS) justified its refusal by the lack of sufficient data proving the effectiveness of the treatment, a decision which raises concerns about the continuation of treatments for the patients concerned.
 Sophie de Duiéry
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29/10/2024 | 
