New ALS Treatment, AMX0035, Lacks Evidence of Benefit, FDA Panel Finds

The main goal was to slow down the fall by a 48-point ALS scale assessment of 12 physical abilities, including walking, speaking, swallowing, dressing, handwriting, and breathing.

“This is the first time we’ve seen improvements in function and survival in an ALS clinical trial,” said Dr. Sabrina Paganoni, Principal Clinical Trial Investigator and Neuromuscular Medicine Specialist at the Healy Center for ALS at Massachusetts General Hospital.

“If access is delayed, patients in my clinic today may never get the time and features they might otherwise have. Access delay is not a risk we should take,” said Dr. Paganoni.

However, FDA reviewers have identified many problems with the phase 2 clinical trial and the open extension. They said in information documents that the benefit found was “borderline statistically significant and may not be conclusive enough to allow determination of efficacy based on a single study.”