When patients are battling a terminal illness and want access to an experimental drug, how much evidence that it works should regulators require before approval?
That’s the question behind many of the Food and Drug Administration’s toughest decisions, including last year’s controversial approval of Aduhelm. Many experts — including the agency’s own outside advisers — say that Alzheimer’s drug is unlikely to help patients.
Less than a year later, the agency may soon approve another drug for a deadly neurodegenerative disease based on partial data that’s being debated by experts. The FDA meets next week to publicly review evidence from a small, mid-stage study of Amylyx Pharmaceuticals’ drug for ALS, or amyotrophic lateral sclerosis.
