“This health Europe that protects us”, an example of Charcot disease

LThe European Medicines Agency (EMA) protects patients with amyotrophic lateral sclerosis (ALS). Despite the technicalities, the topic deserves the attention of the general public. Let’s get back to the facts. American company Amylyx Pharmaceuticals has developed an experimental treatment called “Relyvrio” in the United States and “Albrioza” in Europe to slow the progression of amyotrophic lateral sclerosis. Also called “Charcot disease”, it is a rare orphan neurodegenerative disease that affects motor neurons. This causes progressive paralysis of all muscles. This disease is incurable and fatal today.

After in vitro studies, a clinical trial on a small number of patients with amyotrophic lateral sclerosis was conducted by Amylyx Pharmaceuticals, which studied the effects of albriose. These studies, called “phase 2”, are intended to assess the tolerability of the treatment and to provide an initial idea of ​​its effectiveness. They are usually not enough to show the effectiveness of the medication. There are exceptions when the drug has a dramatic effect, but these cases are rare. The results of this study, called Centauri, were published in 2020. The benefit of Albriosa appeared modest and tolerability was good.

The procedure in these cases is to confirm the result on a large number of patients in a so-called “phase 3” study, which aims to convincingly demonstrate the drug’s effectiveness. In the context of an incurable disease, the scientific and especially the ethical interest of this phase 3 study can be discussed if the first results are sufficient. In this context, Amylyx has filed marketing applications in the United States with the Food and Drug Administration (FDA) and in Europe with the EMA. Authorization was granted by the FDA in September 2022, but the latter had reservations about the effectiveness of the drug and recommended the continuation of the Phase 3 study, which was ongoing.

Conversely, the EMA considered in October 2023 that the data were not sufficient to grant marketing authorisation. The decision drew criticism, particularly from the ALS Research Association (Arsla) in France. The latter wrote several press releases and open letters to protest this decision. These press releases indicate that the EMA is preventing French patients from accessing effective treatments, while these treatments are authorized in the United States. Basically, a technocratic European institution out of touch with the reality of patients will prevent European consumers from accessing an effective product that other countries use. The treatment received early approval in France in October 2023.