Question 1917 — diseases

Summary

Mr. Bérenger Cernon alerts the Minister of Health and Access to Care about the refusal of early access to Qalsody treatment for people suffering from amyotrophic lateral sclerosis (ALS) linked to a mutation in the SOD1 gene. This decision by the High Authority for Health (HAS), rendered on October 10, 2024, deprives these patients of their only hope in the fight against this devastating disease. ALS SOD1 is a rare scourge, affecting 30 to 40 people per year in France, which leaves only 2 to 5 years of life expectancy for diagnosed patients. Qalsody, or Tofersen, is the only existing treatment that directly targets the genetic cause of this form of ALS. While other countries (such as Germany, Italy and the United States of America) authorize and reimburse this treatment, France has just refused both its reimbursement and early access for new patients. This refusal arouses incomprehension: the European Medicines Agency (EMA) has authorized its marketing in February 2024 and in France, the feedback from treated patients is encouraging. Access to Qalsody, authorized by the National Agency for the Safety of Medicines and Health Products (ANSM) since February 2022, shows favorable effects: stability of respiratory, nutritional and motor functions without adverse effects major. The New England Journal of Medicine also published results that demonstrate the biological benefits of the treatment, including the reduction of SOD1 protein levels and neurofilaments in the blood, a crucial marker of ALS prognosis. Why ignore such promising results? This refusal seems all the more unfair as this decision is based on unsuitable criteria, applied in an identical manner for the rare diseases and more common diseases, without taking into account the specific constraints of ALS research. It is unacceptable that French patients are forced to cross borders to access treatment. The move also sends a dire signal to researchers and companies investing in rare diseases, potentially discouraging innovation. France, a pioneer country in field of rare pathologies, is preparing to abandon its leadership role. ALS patients and their families deserve better than to see their country forgo a crucial innovation. Hope is there and it is up to the Minister to act so that they can seize this chance. He therefore asks him to intervene to change this decision.

Machine translation from French. The official text remains authoritative.

Timeline

No timeline events have been ingested for this record yet.

Votes

No vote records are attached yet.

Versions

No version snapshots stored. Document URLs remain at the source.

Documents

No documents linked.

Sponsors

No sponsors or actors listed by the source.

Related records

No cross-record relationships stored yet.