Despite concerns, FDA panel reverses course on ALS drug and recommends approval : Shots - Health News In March, experts who advise the FDA questioned the efficacy of an experimental new drug for ALS. In September, they voted to approve it anyway.

FDA seems poised to approve a new drug for ALS, but does it work?

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Earlier this year, the Food and Drug Administration voted against approving a new medication for Lou Gehrig's disease, or ALS. Now, the FDA appears ready to approve it. The thing is, it's still not clear whether the drug works. NPR's Jon Hamilton reports.

JON HAMILTON, BYLINE: The drug is called AMX0035. And in March, its short-term prospects looked bleak. The drug's maker, Amylyx, was seeking FDA approval based on a single study of 137 patients with ALS. The rare neurological disorder gradually renders a person unable to walk, talk or even breathe. But at a public meeting, members of an FDA advisory committee said they were unimpressed by the drugmaker's study, called CENTAUR.


CALEB ALEXANDER: ...Many features of CENTAUR that limit its persuasiveness.

BRYAN TRAYNOR: The applicants haven't provided robust evidence...

LIANA APOSTOLOVA: The data isn't as strong as we would hope.

THOMAS MONTINE: It did not meet the threshold of...

KENNETH FISCHBECK: Problematic study...

ROBERT ALEXANDER: ...Doesn't establish that this drug is effective in the treatment of ALS.

HAMILTON: Those were the voices of doctors Caleb Alexander, Bryan Traynor, Liana Apostolova, Thomas Montine, Kenneth Fischbeck and Robert Alexander. All six voted no when asked whether the evidence showed the drug was effective. Four other committee members voted yes. Typically, that would have been the end of it until Amylyx had more data. But Neil Thakur of the ALS Association, which helped fund the study, says patients and family members took to the internet.

NEIL THAKUR: There were thousands of emails that went to the commissioner's office. There were over 1,100 comments that went to the advisory committee themselves. And also, there was a sustained effort from ALS clinical and scientific leaders.

HAMILTON: The Amylyx drug combines two existing products. One is a dietary supplement. The other is a prescription drug for a rare metabolic disorder. The combination is meant to slow down the destruction of cells in the brain and spinal cord. And in the Amylyx study, it appeared to extend patients' lives by several months. Thacker says that study does have flaws. He thinks the advisory committee should have been willing to overlook them.

THAKUR: They were asking to hold that drug to the same standard they would hold any drug for any disease that wasn't fatal and had lots of effective treatments.

HAMILTON: Right now, ALS patients have just two options for drug treatment. And even with these drugs, they typically die within 2 to 5 years after a diagnosis. The email campaign by ALS patients seemed to have an effect on some FDA officials. Early this month, the agency took the unusual step of reconvening its advisory committee to reconsider the Amylyx drug. And Thacker of the ALS Association says, this time, the FDA urged committee members to take a different perspective.

THAKUR: This committee - it was clear that they were being asked to make a decision, taking into account the available treatments and the needs of the ALS community.

HAMILTON: The committee also got some additional data on patients in the Amylyx study. And when the committee held its second public hearing, they heard from Dr. Billy Dunn, who directs the FDA's Office of Neuroscience. Dunn suggested that the agency was open to approving the drug.


BILLY DUNN: For these serious diseases, like ALS and so many other neurological conditions, the maximum degree of regulatory flexibility is operational.

HAMILTON: The FDA even revised its question to the committee. Instead of asking whether the drug was effective, they simply asked whether it should be approved. This time, 7 of 9 committee members voted yes. Holly Fernandez Lynch teaches medical ethics and law at the University of Pennsylvania.

HOLLY FERNANDEZ LYNCH: They were swayed by this concern that they might be making the wrong judgment if they recommended the FDA not to approve this product. But nobody, as far as I heard, said this drug meets the substantial evidence standard.

HAMILTON: The usual standard for determining that a drug works. The substantial evidence standard was also in question during consideration of the controversial Alzheimer's drug, Aduhelm. The FDA approved that drug last year, despite an overwhelming no vote from the advisory committee. Lynch says approving the new ALS drug could send a troubling message to pharmaceutical companies.

FERNANDEZ LYNCH: The message that is relayed to companies is you don't actually have to prove your drug works. You have to do the bare minimum to show that maybe it might work.

HAMILTON: An FDA decision on the ALS drug is expected by the end of the month.

Jon Hamilton, NPR News.


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