FDA panel skeptical of controversial ALS drug ahead of vote | Health, Medicine and Fitness
TUESDAY, Sept. 6, 2022 (HealthDay News) — A U.S. Food and Drug Administration panel will again consider approval of an investigational drug for ALSa rare second review for a disease that has no cure.
The same panel that will meet on Wednesday vote last March not to approve the drug for the fatal neurodegenerative disease also known as Lou Gehrig’s disease.
But getting the drug, known as Albrioza (AMX0035), approved is a rallying cause for patients, their families and members of Congress, the Associated Press reported.
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Yet federal regulators said in a information document filed Friday that the company’s new evidence was not “sufficiently independent or compelling” to establish efficacy.
The paper said experts could consider “unmet need in ALS” and the flexibility the agency has to seek approval for drugs that treat life-threatening conditions.
This suggests “there is a chance the FDA is still looking for a way to approve the product,” SVB analyst Marc Goodman wrote in a note to investors. Goodman thinks the drug has about a 50% chance of being approved, the PA reported.
When regulators first reviewed the drug in March, they voted against it 6 to 4 after finding the data was not convincing that it would benefit people with the disease. The panel gave the agency until September 29 to review any further data submitted by the company.
Canadian regulators have already approved the drug for ALS patients, which puts the FDA in a “precarious position,” bioethicist Holly Fernandez-Lynch told the PA.
“They generally like to be ahead when making approval decisions,” said Fernandez-Lynch, who teaches at the University of Pennsylvania. “They like to argue that they’re not a barrier to patients getting things that might help them.”
Amylyx Pharmaceuticals, which makes the drug, said it collected follow-up data on the study which concluded the drug extended the lives of ALS patients by about 10 months.
The drug combines a dietary supplement used in traditional Chinese medicine with an existing prescription medication for liver disorders. The combination protects the cells from premature death, according to Amylyx.
Approval of the combination drug could force insurers to cover it.
But the trial had missing data and implementation errors, according to FDA reviewers. It “still suffers from the same interpretability issues,” they wrote.
ALS patients eventually stop walking, talking, swallowing, and breathing, and typically die within three to five years of diagnosis, as the disease methodically destroys nerve cells.
External contributions will come from patient groups and advocacy groups, including I AM ALS, founded by patient Brian Wallach. Wallach takes the food supplement for the treatment.
“Patients are doing their homework – we know this isn’t going to cure us,” said Wallach, who was diagnosed with ALS in 2017 and spoke through an interpreter. PA. “But we also know it could keep us here until the next drug comes and that could be a cure.”
The US National Institute of Neurological Disorders and Stroke has more on ALS.
SOURCE: US Food and Drug Administration, Briefing Paper, September 2, 2022; Associated Press