Reversing Course, FDA Committee Backs Experimental ALS Drug
News outlets report that the FDA is not required to follow the panel's advice on the drug, developed by Amylyx Pharmaceuticals, but that is the likely outcome. The decision, in a 7-2 vote Wednesday from the FDA's Peripheral and Central Nervous System Drugs Advisory Committee, came as a reversal from a vote against the drug in March.
Stat:
In Reversal, FDA Advisers Vote To Support Approval Of Amylyx's Drug For ALS
At the end of an unusual and dramatic meeting on Wednesday, an independent panel of advisers to the Food and Drug Administration recommended the approval of a new drug to treat people with ALS developed by Amylyx Pharmaceuticals. (Feuerstein, 9/7)
AP:
FDA Panel Backs Much-Debated ALS Drug In Rare, 2nd Review
The Food and Drug Administration advisers voted 7-2 that data from Amylyx Pharma warranted approval, despite hours of debate about the strength and reliability of the company’s lone study. The FDA is not required to follow the group’s advice, but its positive recommendation suggests an approval is likely later this month. The FDA has approved only two therapies for the disease, amyotrophic lateral sclerosis, or ALS, which destroys nerve cells needed for basic functions like walking, talking and swallowing. (Perrone, 9/7)
The Boston Globe:
In A Surprise, FDA Committee Recommends ALS Drug By Cambridge Company, Reversing Previous Vote
The unexpected reversal, which was partly due to pleas from patients and their families and partly due to additional data presented by Amylyx, bodes well for the company. But it is only a recommendation. FDA regulators have the final say in authorizing or rejecting the drug by the end of the month. (Cross, 9/7)
The Wall Street Journal:
Amylyx’s ALS Drug Backed By FDA Advisers
“To deprive ALS patients of a drug that might work—it’s not something that I’ll feel terribly comfortable with. In the previous meeting it wasn’t that clear,” said Liana Apostolova, a neurology professor at the Indiana University School of Medicine, who voted for the drug. Other panel members said the evidence for the drug’s effectiveness was weak. “We essentially have a single study with many nontrivial scientific concerns,” said Caleb Alexander, a professor of epidemiology and medicine at Johns Hopkins Bloomberg School of Public Health, who voted against the drug. (Whyte and Walker, 9/7)
NBC News:
In Reversal, FDA Panel Votes To Recommend Experimental ALS Drug
Mark Weston, the panel’s patient representative, who voted in favor of the drug both times, said that he was “disappointed” with the lack of persuasive data, but noted the unmet need for better treatments for the disease. Dr. Kenneth Fischbeck, a neurologist at the National Institutes of Health, said the company's new analysis contained no new data. (Lovelace Jr., 9/7)
