Question 2141 — pharmacy and medications

Summary

Ms. Anne Le Hénanff draws the attention of the Minister of Health and Access to Care to access to innovative treatments for rare diseases and more particularly to the recent refusal of the High Authority for Health (HAS) to authorize early access to Qalsody treatment in the context of Charcot disease. Qalsody, produced by the Biogen laboratory, targets a specific genetic mutation (SOD1) present in approximately 1.6% of patients suffering from Charcot disease in France, i.e. between 60 and 160 people. This therapy was approved by the Food and Drug Administration (FDA) in the United States of America in April 2023 and by the European Medicines Agency (EMA) in February 2024 and is based on antisense oligonucleotide technology aimed at reducing levels of neurofilaments, biomarkers associated with neuronal damage. However, the HAS refused authorization of early access in October 2024, considering that the available data were not sufficient to guarantee a substantial effect on the progression of the disease. Despite this 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 desire to take into account all new clinical data that could be submitted to it. This decision by the HAS sparked a strong reaction from the Association for Research on ALS (ARSLA), which calls for a review of the decision and 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 ones 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. Also, the MP would like to know what national strategy for the care of ALS patients the Government intends to implement. She also wishes to know what the Government's position is on access and support for innovative treatments for rare diseases.

Machine translation from French. The official text remains authoritative.

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