An FDA panel narrowly sides against an experimental ALS drug
The vote by the agency's health advisers is a potential setback for patient groups who have lobbied for the medication’s approval for more than a year.
U.S. health advisers have narrowly ruled against an experimental drug for the debilitating illness known as Lou Gehrig’s disease.
Advisers to the Food and Drug Administration voted 6-4 that a single study from Amylyx Pharmaceuticals failed to establish the drug’s effectiveness in treating the deadly neurodegenerative disease ALS, amyotrophic lateral sclerosis.
Wednesday's vote is a potential setback for patient groups who have lobbied for the medication’s approval for ALS for more than a year.
A majority of panelists agreed with a negative review published by FDA scientists earlier in the week. Some said they hoped results of a larger study now underway would provide more evidence of the drug's effectiveness. T
he panel's vote is nonbinding, and the FDA will make its final decision by July.
ALS destroys nerve cells in the brain and spinal cord needed to walk, talk, swallow and — eventually — breathe. There is no cure and most people die within three to five years of their first symptoms.