The primary goal was to slow the decline. 48-point ALS scale Rate 12 physical abilities, including walking, speaking, swallowing, dressing, handwriting and breathing. Over the 24 weeks, patients who took the placebo dropped 2.32 points more than those who took the drug combination, resulting in a 25 percent slower decline in the treated patients over that time.
The open-label extension study included 90 patients, including 34 from the placebo group, who started taking AMX0035 approximately seven months after those who received it from the start. The researchers reported to the FDA that those who received the treatment the longest spent an average of 4.8 months longer before being hospitalized, placed on a ventilator, or died.
D., principal investigator of the clinical trial and neuromuscular medicine specialist at the Massachusetts General Hospital Healey ALS Center. “This is the first time we’ve seen a benefit in terms of both function and survival in an ALS clinical trial,” said Sabrina Paganoni.
“If access is delayed, patients in my clinic today may never get the time and function they can have. Delaying access is not a risk we should take,” said Dr. paganoni
But FDA reviewers identified many issues with both the Phase 2 clinical trial and the open-label extension. they said briefing documents He said the benefit identified “is bordering on statistically significant and may not be convincing enough to allow a determination of efficacy based on a single study.”
The leader of the FDA team, Dr. Emily Freilich said at the hearing that the clinical trial results suggesting that the drug slowed the decline in ALS functional scale were “not very convincing” and that the trial’s secondary measures — including muscle strength, breathing — were “not very convincing.” ability and whether patients were hospitalized – did not “generally support” benefit.
FDA officials said even the evidence from the extension study did not show that the treatment could help patients live longer. Patients who took placebo and never switched to AMX0035 survived a median of 1,295 days, while patients who took AMX0035 for longer than 96 weeks survived an average of 1,237 days.