FDA approves Amylyx's much-debated drug for ALS

The Food and Drug Administration approved Amylyx's highly debated drug for ALS disease on Thursday, September 29, 2022, based on results from a small-scale mid-stage trial, in which patients with the disease seemed to progress more slowly and survive several months longer.

This development is kind of thanks to the 2014 trend called " the Ice Bucket Challenge," which encouraged people to donate to the ALS Association or dump an ice-cold bucket of water over their heads. According to a news report published by the ALS Association, the initiation raised $115 million, $2.2 million of which was used for funding the study testing the recently authorized drug Relyvrio.

The trial included 137 people

Amyotrophic lateral sclerosis, or ALS in short, is a progressive neurodegenerative disease that harms nerve cells in the brain and spinal cord. Also known as motor neuron disease (MND) and Lou Gehrig's disease, it leads to loss of muscle control.

There is no known treatment for the disease, yet, and most patients live only two to five years after being diagnosed.

The clinical trial included 137 people with ALS disease and made them test Amylyx’s drug called Relyvrio. The results have demonstrated that subjects who took the drug lived nearly ten months longer than those who didn’t take it and the drug seemed to delay hospitalizations.

Concerns about the persuasiveness

With the FDA's approval of the drug, Relyvrio has become the third FDA-approved medicine for ALS. It is surprising because typically, the agency requires two large studies or one study with extremely compelling survival results before approving a drug.

“It’s hard to put into words how significant this approval is for the thousands of Americans who are living with ALS and their families,” said Larry Falivena, a member of the ALS Association Board of Trustees who was diagnosed with ALS in 2017. “The ALS community has proven that our advocacy can impact decisions that are being made about our health. We need new treatments as quickly as possible if we are going to turn ALS into a livable disease and eventually cure it.”

When the medicine was brought before an FDA advisory committee for the first time in March, questions about the medicine and the trial were also raised. Agency experts questioned the Amylyx trial's persuasiveness in briefing documents published before the meeting. By a slim margin, the committee decided against recommending rejection.

However, the FDA took a surprising step by calling for a second advisory committee just six months later after Amylyx shared a second analysis of its trial results. The advisory committee changed its mind about the medicine during that meeting and decided to approve it, according to a report by NBC News.