Charcot disease: a controversial treatment withdrawn in the United States and Canada

2024-04-04 19:53:47

A treatment for Charcot disease will be withdrawn from the market in the United States and Canada following inconclusive results during a clinical trial, Amylyx Pharmaceuticals announced Thursday.

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The American health authorities had chosen to approve it in 2022 on the basis of a first smaller clinical trial, that is to say on a limited number of patients.

They made this decision in view of the seriousness of this disease, for which there are few treatments and which today remains incurable.

Amyotrophic lateral sclerosis (ALS), or Charcot disease, results in a progressive paralysis of the muscles which gradually makes it impossible to walk, eat, breathe or speak, creating a state of confinement for the patient whose brain and intellectual abilities remain intact.

Once diagnosed, life expectancy is generally three to five years.

“Amylyx has begun the process” to “voluntarily withdraw sales authorizations” for the treatment, AMX0035, in partnership with the United States Medicines Agency (FDA) and Health Canada, the pharmaceutical company said in a press release.

The treatment in question, which aimed to slow the progression of the disease and was marketed under the name Relyvrio in the United States and Albrioza in Canada, “will no longer be available to new patients from today”, he said. she added.

This is a “difficult time for the ALS community,” its bosses, Joshua Cohen and Justin Klee, acknowledged in a statement.

For patients already under treatment (around 3,900 in the United States), the American company said it was offering to continue, free of charge.

Handle of treatments

“We commend Amylyx for its decision to remove Relyvrio from the market, while ensuring that people living with ALS can access the drug if they believe it helps them,” commented the ALS Association.

This American organization had pushed for the authorization of the treatment, which was then based on a single study carried out on 137 patients.

Last month, Amylyx Pharmaceuticals announced the results of a trial carried out this time on 664 participants, showing that the treatment made no difference compared to a placebo.

Its authorization in the United States, but not in Europe, had provoked the anger of patients in France in particular.

The European Medicines Agency (EMA) considered that the preliminary results did not convincingly demonstrate a slowdown in the progression of the disease.

Amylyx Pharmaceuticals, of which this treatment was the only one marketed, announced Thursday that it would reduce its workforce by 70% and concentrate on research into other drugs.

There are only a handful of approved treatments for CD in the United States, where an average of 5,000 new people are diagnosed with it each year.

The latest, Qalsody, from the American laboratory Biogen, only concerns a portion of patients with a certain genetic mutation. Riluzole, authorized since 1995, can extend patients’ lives by around three months.

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