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Panel Not Convinced on ALS Treatment

A US Food and Drug Administration panel finds there is not enough evidence showing that a new treatment for amyotrophic lateral sclerosis is effective, the New York Times reports.

It adds that the treatment, dubbed AMX0035, is a combination of two existing drugs, taurursodiol and sodium phenylbutyrate, a mixture that was thought up by Justin Klee and Joshua Cohen while still students at Brown University. They suspected that the two drugs together could help protect neurons, the Times adds, noting that the two went on to found Amylyx Pharmaceuticals.

The Times writes that the FDA usually requires two phase 3 studies to support approval, though does sometimes allow one such study with additional supporting evidence if the disease is severe with few treatment options. It notes that the Amylyx data comes from a small phase 2 study, with more data from an open-label extension study. That study found patients who received AMX0035 had a 25 percent slower decline than those who received placebo, the Times adds.

The panel, though, did not find the data persuasive, it says, particularly as the patients who received the treatment did not appear to live longer than those who did not.

As the Times notes, this comes after the FDA came under criticism for approving Biogen's Aduhelm to treat Alzheimer's disease, which also had limited evidence supporting its efficacy.