Morning Briefing
Summaries of health policy coverage from major news organizations
Reversing Course, FDA Committee Backs Experimental ALS Drug
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)
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鈥檚 lone study. The FDA is not required to follow the group鈥檚 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 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)
鈥淭o deprive ALS patients of a drug that might work鈥攊t鈥檚 not something that I鈥檒l feel terribly comfortable with. In the previous meeting it wasn鈥檛 that clear,鈥 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鈥檚 effectiveness was weak. 鈥淲e essentially have a single study with many nontrivial scientific concerns,鈥 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)
Mark Weston, the panel鈥檚 patient representative, who voted in favor of the drug both times, said that he was 鈥渄isappointed鈥 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)
