Qalsody, a pending hope for ALS patients
Qalsody, produced by the Biogen laboratory, targets a specific genetic mutation (SOD1) present in approximately 1.6% of patients with Charcot disease in France, or between 60 and 160 people. This therapy was approved by the Food and Drug Administration (FDA) in the United States in April 2023 and by the European Medicines Agency (EMA) in February 2024. It is based on antisense oligonucleotide technology aimed at reducing levels of neurofilaments, biomarkers associated with neuronal damage.
However, the HAS refused early access authorization in October 2024, arguing that the data from the phase 3 clinical study, published in 2022 in the New England Journal of Medicinedid not demonstrate significant benefits on the clinical progression of ALS. The results of this study revealed no notable benefit on the ALS Functional Scale (ALSFRS-R), the primary measure of clinical effectiveness.
Controversial effectiveness criteria and ongoing evaluation
The refusal of early access is mainly based on two criteria that were not met by Qalsody: demonstrated clinical benefit and improved care for patients. The HAS considered that the available data are not sufficient to guarantee a substantial effect on the progression of the disease. Indeed, although Qalsody showed an impact on biological markers, notably the reduction of neurofilaments, this improvement did not translate into a tangible clinical benefit at the end of the 28 weeks of the clinical trial.
Despite this initial decision, HAS does not exclude the reimbursement of Qalsody as part of another traditional application procedure. Discussions between HAS and Biogen continue for a new review. In this context, HAS expressed its willingness to take into account any new clinical data that may be submitted, offering a glimmer of hope to patients and families affected by this devastating disease.
Public debate and growing pressure from patient associations
The HAS decision sparked a strong reaction from the Association for Research on ALS (ARSLA), which considers the decision “cruel and unacceptable” for French patients. Faced with this situation, the association launched a petition addressed to President Emmanuel Macron, calling for a review of the decision and for support for Qalsody, already available in other European countries such as Germany and Italy. .
This debate highlights the complex issues of access to innovative treatments for rare diseases. The next few weeks will be decisive, because the new opinion from the HAS could influence the national strategy for the management of ALS patients and potentially allow access to this treatment for people in therapeutic impasse.
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