The ALS Association, known for its Ice Bucket Challenge fundraising, is pushing for a speedy FDA approval of Amylyx’s now-pending ALS candidate submission—but is history repeating itself?
In the wake of Biogen’s Aduhelm surprise approval this summer, some are questioning what role that approval is playing in the FDA’s renewed willingness to evaluate Amylyx’s drug. Is the FDA heading down a new path of bending to public pressure and advocacy?
The leading ALS advocacy group is unabashedly pushing for a “swift” FDA approval of the drug for all ALS patients after an active year of lobbying. It submitted a petition with 50,000 signatures and held an emotionally charged “We Can’t Wait” action meeting, with more than 10 people who have ALS speaking directly to FDA leaders including Patrizia Cavazzoni, the director of the Center for Drug Evaluation and Research (CDER).
While ALS Association Neil Thakur chief mission officer couldn’t speak to a possibly changing FDA mindset, it is clear the agency changed its tune about Amylyx’s candidate after the association lobbied for the agency to be consistent in adhering to its own 2019 ALS clinical trial guidance and its associated regulatory flexibility.
In June, the association publicly called out the FDA’s stated commitment in a blog post pointing to the case of Biogen and Aduhelm.
“Just recently, the FDA used its regulatory flexibility to approve Aduhelm for the treatment of Alzheimer’s disease. This is good news for people living with Alzheimer’s and their loved ones. But we are left to wonder [why\ the FDA is not using similar flexibility for a promising ALS treatment with strong safety data that provides clinically meaningful benefits. People with ALS and their loved ones cannot wait,” the group said.
Thakur noted, however, that his organization had already detected a change in the FDA’s position before the Aduhelm nod.
“By the time of the (We Can’t Wait) meeting in May, the FDA had come around to recognizing that this was something they had to move forward on,” he said, adding that bolstering its case was Canadian and EU drug evaluators pressing ahead on the Amylyx candidate.
Thakur isn’t apologetic about the ALS Association’s role in trying to get the drug approved.
“Our job as advocates for the ALS community is to end the disease. If there’s a drug that’s going to help do that, then we’re going to push forward—and if there’s a drug on the market that’s not going to do that, we need to find a way to take that drug off the market. It goes both ways,” Thakur said.
He emphasized the high safety profile of the Amylyx drug as well as its “modest positive effect” for ALS patients.
Thakur is also realistic. The drug is not a silver bullet; however, the ALS Association’s intention in backing it, and other similar drugs, is for “incremental gains and [to] increase their life span until we figure out some really solid ultimate cures,” he said.
For now, the ALS Association plans to wait and see what happens after Amylyx files for FDA approval, but it won’t hesitate to push again if the FDA slow rolls the process.
“Our community will be very concerned. There really is no way to do this fast enough for ALS—it just has to be fast, fast, fast,” he said.