Controversial treatment for Charcot disease has been canceled in the US and Canada.

A drug to treat Charcot’s disease will be withdrawn from sale in the United States and Canada after inconclusive clinical trial results, Amylyx Pharmaceuticals said. In 2022, US health authorities decided to approve it based on a first, small clinical trial conducted on a limited number of patients.

They made this decision because of the seriousness of the disease, for which there are few treatments and which remains incurable today. Amyotrophic lateral sclerosis (ALS), or Charcot’s disease, causes progressive muscle paralysis that gradually makes it impossible to walk, eat, breathe or speak, creating a state of isolation for the patient whose brain and intellectual abilities remain intact. Once diagnosed, life expectancy is usually three to five years.

No difference compared to placebo

Amylyx has begun the process of “voluntarily withdrawing the marketing authorization” of AMX0035 in partnership with the U.S. Drug Administration (FDA) and Health Canada, the pharmaceutical company said. This treatment aimed to slow the progression of the disease and was sold under the name Relyvrio in the US and Albrioza in Canada. The lab added that as of today it will “no longer be available to new patients.”

For patients already on treatment, of which there are about 3,900 in the US, the US company said it is offering to continue treatment free of charge. “We commend Amylyx for its decision to remove Relivrio from the market while ensuring that people living with ALS can access the drug if they believe it helps them,” the ALS Association commented.

Last month, Amylyx Pharmaceuticals announced the results of a study, this time involving 664 participants, which showed the treatment made no difference compared to a placebo.

Authorized for a certain period of time in accordance with French conditions.

The approval of the treatment in the US, but not in Europe, has angered patients, particularly in France. But the European Medicines Agency (EMA) said the preliminary results did not convincingly demonstrate a slowing of disease progression.

However, last year French health authorities finally approved it under certain conditions. But after the results were published in March, this so-called “compassionate” access was brought to an end, a spokesman for the National Agency for the Safety of Medicines (ANSM) said.

As a result, Amylyx Pharmaceuticals announced it would cut its workforce by 70% and focus on research into other drugs. Amylyx instantly lost 80% of its stock value, according to the Washington Post.