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Last July, the American ALS Association and Yo Soy ALS petitioned the US Food and Drug Administration (FDA) to approve one of the most promising treatments for Amyotrophic Lateral Sclerosis (ALS): AMX0035of the pharmaceutical company Amylyx. His request was supported by phase 2 results of AMX0035 published in The New England Journal of Medicine that showed the drug slowed the progression of ALS.
However, a panel of outside FDA advisers voted Wednesday against approving the drug, saying clinical trial data submitted by the company failed to establish that it was effective against the disease.
AMX0035 is an oral therapy dissolved in water that combines two existing drugs, sodium phenylbutyrate and taurursodiol, which prevent nerve cell death.
ALS is a heterogeneous disease in many aspects, since it can be start any age, can affect any muscle group, and has a highly variable survival rate. In any case, the average age of onset is between 60-69 years; in almost the majority of patients the disease begins in the muscles that control speech, swallowing and chewing or in the muscles of the extremities (only a small group of cases have a generalized or respiratory onset); and approximately 50% of patients die within 3 years of diagnosis.
It’s about the third most common neurodegenerative disease in Spain in incidence, after dementia and Parkinson’s disease. However, its high mortality means that, according to estimates by the Spanish Society of Neurology (SEN), there are currently some 3,000 affected in Spain, although 3 new cases are detected every day.
This decision is not binding, although the agency generally follows the panel’s recommendations, although it is not required to do so. The final decision of the FDA It is expected on June 29.
The panel’s decision was made based on documents released by the FDA expressing concern about the data and efficacy of the drug trial.
“We were asked to persuasively and robustly pursue substantial evidence, and I think this trial falls short of that goal,” says Kenneth Fischbeck, one of the panel members and an investigator at the US National Institutes of Health.
This decision contrasts with the strong support expressed by patient associations who point out that the drug “does no harm.”
Amylyx recently started a new trial with more patients, around 600 from all over the world, whose results are expected by 2024.
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