The odds that Amylyx Pharmaceuticals wins approval from the Food and Drug Administration for Albrioza, its treatment for ALS, have increased significantly over the past two weeks. My prediction: The FDA grants accelerated approval to Albrioza, with the requirement that Amylyx confirm the drug’s benefit for patients with results from the ongoing Phase 3 study — data expected in 2024.
I’m capable of making a positive prediction occasionally. I’m not all doom and gloom. This one feels right, with echoes of how the FDA acted on eteplirsen, the Duchenne muscular dystrophy drug, in 2016, and more recently, Biogen’s Alzheimer’s treatment Aduhelm, in 2021.
Back in March, agency reviewers worked hard to raise doubts about Albrioza (technically, AMX0035 because it’s still experimental in the U.S.) to a panel of outside advisers. It was clear that Billy Dunn, the agency’s chief neuroscience regulator, did not want to approve the Amylyx drug, based mostly on an argument that the company’s randomized, placebo-controlled study wasn’t sufficiently persuasive from a statistical standpoint, despite achieving its primary efficacy goal of slowing disease progression. But even with all that work against Albrioza, Dunn and his colleagues only got a 6-4 negative vote from the expert panel, and one of those experts, a biostatistician from the National Institutes of Health, voted to recommend the drug’s approval.
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