FDA panel narrowly sides against experimental ALS drug

U.S. health advisers have narrowly ruled against an experimental drug for the debilitating illness known as Lou Gehrig’s disease

WASHINGTON -- Federal health advisers on Wednesday narrowly ruled against an experimental drug for the debilitating illness known as Lou Gehrig’s disease, a potential setback for patient groups who have lobbied for the medication’s approval.

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.

“I think it would be a disservice to the patients and their families to approve a treatment that is of uncertain benefit,” said Dr. Kenneth Fischbeck of the National Institutes of Health. “It gets in the way of developing truly effective treatments if it turns out not to be effective."

Fischbeck and other panelists said they hoped results of a larger study now underway would provide more evidence.