This $158,000 Drug Likely Doesn’t Work. Why Was It Approved?
ALS patients are desperate for better treatments. Source: Getty Images
Hi, it’s Bob in New York. A $158,000 ALS drug was approved based on paltry evidence. Now it turns out it likely doesn’t work. But first...
When Amylyx Pharmaceuticals Inc. snagged US approval for a $158,000-a-year drug to treat amyotrophic lateral sclerosis, it was a controversial decision. The company had completed only one small efficacy trial in 137 patients. The first time the drug went before a Food and Drug Administration advisory panel in March 2022, the panel voted 6-4 that the drug had not been proven to be effective.
The FDA, under pressure from patient groups to approve the medicine, took the unusual move of convening a second panel hearing on the same drug less than six months later. At the second hearing, Amylyx co-founder Justin Klee promised to take the drug off the market if it was approved and later studies failed to confirm the medicine’s effectiveness.