FDA Approves Ice Bucket Challenge Drug
This week an ALS drug that has been previously denied by the FDA won approval despite minimal research or data to support its benefits. The FDA increasingly faced mounting pressure for approval once the drug was approved in Canada and patients rallied to get the new drug approved. The ALS Association invested $2.2 million (out of the $15 million raised through the Ice Bucket Challenge) into the development of this drug and stands to make back $3.3 million in drug sales according to the Associated Press.
The ALS Association has been the subject of criticism for its loud and sometimes misguided publicity tactics. For new drug approval, the FDA usually requires two large studies to show the effectiveness of the drug. Amylyx is being approved with one medium-sized study, and the first big study will be complete in 2024. Part of the FDA rationale is that unfortunately, ALS is a terminal diagnosis so something might be better than nothing.
Today about 20,000 Americans have ALS. There have been a handful of questionable FDA decisions recently, one of which is the controversial Alzheimer’s drug Aduhelm which according to AP, “has not yet been shown to slow the disease”. While trying a cure may be better than nothing, for patients and their families these medications are expensive for something not shown to work. This ALS drug, Amylyx, will cost anywhere between $9-$30K for patients, and many patients are already on one or two drugs that cost about the same and other medical bills.