Committee members, who voted 4-6 against Amylyx Pharmaceuticals' drug, said the decision was difficult, given the lack of treatments available for the fatal disease.
The 4-6 vote capped off an all-day meeting where members of the agency’s
"I would not be surprised by a decision in either direction at this point," Holly Fernandez Lynch, an assistant professor of medical ethics at the University of Pennsylvania, said."I think what's clear is that if the drug works, the benefits that it would offer are certainly meaningful to patients." But ultimately, the majority of the committee members said the data showing a slowing in the progression of the disease was not persuasive enough to recommend approval.
They also pointed out problems with how the trial was conducted, such as the first 18 patients receiving the drug instead of being randomly selected to receive the drug or a placebo, which Amylyx attributed to shipping problems. Those patients were still included in the company’s application to the FDA.of the drug, saying in briefing documents that the company’s data on the drug “may not be sufficiently persuasive” to recommend approval.
The benefit of the Amylyx drug is “clear,” said Dr. Sabrina Paganoni, a principal investigator of the smaller trial. “Delaying access is not a risk that we should take.”The committee’s vote is only a recommendation, and the FDA doesn’t have to follow its advice. But the group’s lack of endorsement may weigh heavily on the agency, which has been under intense scrutiny from the scientific community over its handling of recent approvals.
Ireland Latest News, Ireland Headlines
Similar News:You can also read news stories similar to this one that we have collected from other news sources.
FDA panel narrowly sides against experimental ALS drugU.S. health advisers have narrowly ruled against an experimental drug for the debilitating illness known as Lou Gehrig’s disease.
Read more »
FDA panel narrowly sides against experimental ALS drugFederal 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.
Read more »
FDA panel narrowly sides against experimental ALS drugFederal health advisers have narrowly ruled against an experimental drug for the debilitating illness known as Lou Gehrig’s disease
Read more »
FDA advisers to review ALS drug funded by Ice Bucket ChallengeAdvocates are pushing for a positive recommendation from the panel, but a review by FDA scientists said data was lacking on the drug's effectiveness.
Read more »