Against all expectations, the French authorities have refused early access to the drug Qalsody (Tofersen) for patients suffering from amyotrophic lateral sclerosis (ALS) with a mutation of the SOD1 gene. A procedure that has provoked the anger of patients and associations.
Although the drug Qalsody had been approved in recent months by the American and European health authorities and was a source of hope for patients suffering from amyotrophic lateral sclerosis (ALS) with a mutation of the SOD1 gene, the High Authority for Health has decided to refuse early access to this treatment. For the Association for Research on ALS (ARSLA), the main French association fighting the disease, this decision creates a " profound inequality of treatment " for all these patients.
An essential treatment for patients
Amyotrophic lateral sclerosis (ALS) or Lou Gehrig's disease is a neurodegenerative disease that causes progressive paralysis and then death of the patient.
Among the patients, 2% of them are affected by a specific genetic mutation: ALS-SOD1 in which mutations of the SOD1 gene lead to the creation of a toxic form of the SOD1 protein which causes the degeneration of motor neurons.
However, the drug Qalsody , based on the molecule tofersen, is currently " the only treatment targeting the genetic cause of this rare and fatal form of ALS ", states the ARSLA in a press release.
In France, several dozen patients have so far benefited from compassionate access to this drug, authorized in 2022 by the National Agency for the Safety of Medicines (ANSM).
" However, this recent decision, based on clinical assessments deemed insufficient, now leaves them without certainty as to whether to continue their treatment, which is nevertheless essential for them," the organization further explains.
Qalsody: a mixed benefit?
Indeed, French health authorities have rejected the approval of Qalsody, developed by the Biogen laboratory, due to the lack of sufficient data to prove its effectiveness.
More specifically, the study carried out by the international company Biogen and published in 2022 in the New England Journal of Medicine (NEJM), did not demonstrate any significant slowing down of the symptoms of the disease.
" This medicine is not likely to meet an insufficiently covered medical need," confirmed the High Authority for Health (HAS) this week.
Concretely, what does this change for patients?
Lack of early access to treatment deprives patients of the opportunity to obtain reimbursement for this drug.
" Introduced on July 1, 2021, the early access authorization allows patients rapid reimbursed access to drugs that are not yet authorized or covered by common law. These early access authorizations are granted by the High Authority for Health (HAS), following an opinion from the National Agency for the Safety of Medicines and Health Products (ANSM) relating to the presumption of efficacy and safety of products or a marketing authorization," confirms the National Order of Pharmacists.
A refusal, which creates a " precarious situation for patients who had observed stability in their motor and respiratory functions thanks to Qalsody ", further deplores the Association for Research on ALS (ARSLA).
" These patients now find themselves faced with a difficult choice: risk seeing their condition deteriorate or consider solutions abroad, with the constraints and costs that this implies," warns ARSLA, which calls on the authorities to " reconsider this decision."