Statement of Dr. Diana Zuckerman on Behalf of the National Center for Health Research
FDA Advisory Committee Meeting on AMX0035 for ALS
September 7, 2022
I’m Dr. Diana Zuckerman, president of the National Center for Health Research. Our nonprofit center scrutinizes the safety and effectiveness of medical products, and we don’t accept funding from companies that make those products, so I have no conflicts of interest.
My expertise is based on postdoctoral training in epidemiology and public health, as a former faculty member and researcher at Yale and Harvard, and my policy work on FDA issues. I’m currently on the board of the nonprofit Alliance for a Stronger FDA, which educates Congress about the need to financially support the work of the FDA.
ALS is a terrible disease, and what’s needed is a more effective treatment with clinically meaningful benefits. We need better evidence for AMX0035 because if it’s approved, it will undermine 3 randomized-controlled trials of ALS treatments. Even if the U.S. enrollment in the PHOENIX study is completed, that doesn’t mean the follow-up has been completed. And what about placebo-controlled studies of other ALS treatments? Nobody wants to be in a placebo group. Canada’s conditional approval standards are lower than those for FDA full approval, so we shouldn’t be influenced by Canada’s decision.
Sponsors always have the option of voluntarily removing the product from the market, but that rarely happens, and only under very strong pressure from FDA or from lawsuits. In this case, FDA has repeatedly told Amylyx what kind of study design and analyses would be persuasive. Amylyx repeatedly rejected those suggestions, and currently disagrees with FDA’s criticism. Today we all heard Amylyx tell us that they believe their data provide, quote, “a robust confirmation,” unquote, that their drug worked, so it seems naive to think that Amylyx would voluntarily withdraw their drug from the market unless the PHOENIX study results were really terrible, and none of us want that.
What we want is better evidence, and until that’s available, patients deserve free access through clinical trials and through FDA’s expanded access program for experimental drugs.
In conclusion, I know your votes will be tough today. We all wish the company had done a better job of gathering solid evidence, and that’s on Amylyx to fix, not on the FDA to excuse. Meanwhile, isn’t there a data monitoring committee for the PHOENIX study to consider earlier outcome, perhaps in just a few months, so we’ll know more about the effectiveness of this drug?
Thank you very much.