FDA again faces fraught review of sought-after drug

WASHINGTON — 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 the 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 this week to publicly review evidence from a small, midstage study of Amylyx Pharmaceuticals’ drug for ALS, or amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease.

Regulators told Amylyx last year it would need to conduct a large, confirmatory study before seeking approval, according to the company. But after months of intense lobbying by ALS patients and their representatives in Congress, the agency said it could submit the drug based on the smaller study.

The change was so abrupt it surprised even some doctors who helped study the treatment, which appears to modestly slow patients’ decline.

“The effect is there, but it’s not a home run,” said Dr. Jeffrey Rothstein of Johns Hopkins University. “Does it really work? I don’t know. That’s why I’d like to see a second study.”

The FDA traditionally requires two large, late-stage studies for approval. For deadly diseases such as cancer, one study showing promising early results is often accepted. The Amylyx decision comes as government investigators look into Aduhelm’s approval, including whether the agency buckled under pressure from Alzheimer’s groups and pharmaceutical interests.

Experts who study FDA decision-making see a troubling pattern in which the beleaguered agency is continually pressured to accept weaker evidence, damaging its scientific credibility and opening the door to ineffective treatments.

“This is what many people were concerned about in terms of the precedent for FDA approving Aduhelm,” said Dr.

Joseph Ross of Yale University. “They essentially capitulated to both industry and patient advocacy pressure, as opposed to abiding by the science.” An FDA spokesperson declined to discuss the review. FDA will post its initial review of the drug ahead of Wednesday’s meeting.

Kevin Allen / ALS Association Members of the ALS Association meet in Washington this month as part of an effort to lobby federal officials for access to an experimental drug developed by Amylyx Pharmaceuticals.