Federal health regulators remain unconvinced of the benefits of a high-profile experimental drug for a debilitating disease known as Lou Gehrig’s disease, even as they prepare to offer their drugmakers a rare second chance Opportunity to open up for therapy.
Amylyx Pharmaceuticals’ experimental drug has become a cause of solidarity for patients with deadly neurodegenerative diseases, their families and members of Congress Push for FDA approval of the drug.
But regulators said Friday that the drugmaker’s new analysis was “not independent or convincing enough” to establish an effective Sex. The agency released the results of its review ahead of Wednesday’s meeting of outside advisors, who will vote on whether to recommend approval.
In March, the same panel of neurology experts voted 6-4 that the company’s data failed to show a convincing benefit in ALS or amyotrophic lateral sclerosis. FDA calls second review after its advisors vote Meetings are extremely rare.
FDA will ask the panel to review several new statistical analyses, which the company says strengthens Evidence that its drugs can prolong life and delay hospitalizations and other serious complications has led to experts taking into account the “unmet need in ALS,” the severity of the disease and other factors specific to end-stage disease, the FDA said.
Elsewhere in the review, the FDA has detailed the flexibility it can apply to drug approval decisions, especially for fatal diseases, suggesting that “It’s still possible that the FDA is still looking for a way to approve the product,” SVB analyst Mark Goodman wrote in a note to investors, giving Amylyx a 50 percent chance of approval.
ALS destroys the nerve cells needed to walk, speak, swallow, and ultimately – breathe. There is no cure, and most people will experience it within three to five years DEATH.
FDA’s review reflects some of the biggest issues facing the FDA, including: How strict should be the approval criteria for drugs for rare, deadly diseases? How much, if any, should be given to external demands from patients, advocates and their political allies?
Usually, FDA approval requires two large studies or one study with a “very convincing” effect on survival.
Amylyx’s data came from a small study, and the mid-stage trial showed some benefit in slowing the disease, according to FDA reviewers, but due to missing data, implementation errors and Other problems have been compromised.
Follow-up data collected after the study concluded that the drug extended lifespan, Amylyx said. When the company followed patients who continued to take the drug, they lived about 10 months longer than patients who never took the drug, according to a new company analysis.
, but the FDA said Friday that the new approach “faces the same interpretability challenges as the initial Amylyx study” and that the new analysis “is not independent data.”
The FDA did not publicly explain its rationale for the meeting. But some outside analysts believe the agency hopes more outside input will strengthen its influence when it makes a final decision, expected by the end of the month.
Amylyx’s drug is a combination of two older pharmaceutical ingredients: a prescription drug for liver disease and a Chinese medicine-related dietary supplements. The Cambridge, Massachusetts-based company has patented the combination and says the chemicals work together to protect cells from premature death. Its co-founders first discovered the combination among students at Brown University.
Some ALS patients already take both drugs. FDA approval could force insurers to cover the cost of treatment.
FDA will again hear from patients and advocacy groups such as I AM ALS has lobbied FDA and Congress for more than two years to provide this drug. The group’s founder, Brian Wallach, said ALS patients, doctors and researchers felt the company’s data deserved approval.
Their homework — we know it’s not going to cure us,” said Wallach, who was diagnosed in 2017 Has ALS and speaks through a translator. “But we also know it might keep us here until the next drug comes along and may be a treatment. “
Wallach is currently involved in a portion of Amylyx’s treatment, which is available as a dietary supplement.
External developments that may lead to FDA approval.
In June, Canadian regulators approved the drug for ALS patients, the first country to do so. Bioethicist Holly Fernandez-Lynch said the decision put FDA regulators in a “precarious position” “.
“They usually like to make decisions up front when they make approval decisions,” says Fernandez-Lynch, who teaches at the University of Pennsylvania “They like to make the argument that they don’t prevent patients from accessing things that might be helpful to them. “
Amylyx fell more than 23% in Friday’s session to close at $18.
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