By MATTHEW PERRONE, AP Health Writer
WASHINGTON (AP) — A hotly debated drug for Lou Gehrig’s disease won U.S. approval Thursday, a long-sought victory for patients that is likely to renew questions about the scientific rigor behind government drug reviews. experimental.
The Food and Drug Administration approved the drug from Amylyx Pharmaceuticals based on the results of a small, mid-stage study in which patients with the debilitating disease appeared to progress more slowly and survive several months longer. The FDA generally requires two large studies or one study with “very persuasive” survival results for approval.
“This approval provides another important treatment option for ALS, a life-threatening disease that currently has no cure,” FDA neurology drug director Dr. Billy Dunn said in a statement.
The drug, Relyvrio, is the third drug approved in the US for amyotrophic lateral sclerosis, or ALS, which destroys nerve cells needed for basic functions like walking, talking and swallowing.
The FDA review has become a flashpoint in broader debates about the regulatory agency, including how flexible it should be in reviewing drugs for deadly diseases and how much weight it should give to appeals from patients and other outside voices.
“I think it shows the FDA’s ability to be easy and I think it shows a lot of tenacity on the part of ALS patients and advocates,” said Dr. Catherine Lomen-Hoerth, an ALS specialist at the University of California, San Francisco. . “The company really tried to do everything they could to get this potentially promising drug to patients.”
Amylyx’s drug is the latest in a series of neurological drugs that have won FDA approval despite questionable efficacy data. The agency still faces two government investigations into its approval last year of the Alzheimer’s drug Aduhelm, which has yet to be shown to curb the disease.
In an online memo summarizing its decision, the FDA said “regulatory flexibility” was appropriate to approve Relyvrio, “given the serious and life-threatening nature of ALS and the substantial unmet need.”
The latest approval followed a remarkably turbulent path, including two negative reviews from internal FDA scientists, who called the company’s results “borderline” and “not persuasive.” A panel of outside advisers backed that negative view in March, narrowly voting against the drug.
But the FDA has faced intense pressure from ALS patients, advocates and members of Congress. In recent weeks, the agency received more than 1,300 written comments from the ALS community supporting the treatment.
That outpouring helped sway the same panel of experts when the FDA brought them back together last month to review Amylyx’s drug. The second time, they backed the drug, 7-2. The vote was not binding, but appeared to open the door to FDA approval.
Several panelists said they were reassured by an extraordinary exchange at the meeting in which an FDA official requested, and Amylyx stated, that the company would voluntarily withdraw its drug from the market if a large ongoing study did not confirm its benefit.
That 600-patient study is expected to report results in 2024.
But experts have pointed out the many potential problems with such an informal engagement. The FDA and the company may disagree on whether the final data supports the drug; or a company that purchases the drug in the future may not feel bound by Amylyx’s commitment.
The powdered medicine is a combination of two older ingredients: a prescription drug for liver disorders and a dietary supplement associated with traditional Chinese medicine. Cambridge, Massachusetts-based Amylyx has patented the combination and says the chemicals work together to protect cells from premature death.
Some ALS patients already take both drugs separately, sometimes paying $5,000 a month for the prescribed component, according to doctors. The FDA approval is expected to force insurers to cover Amylyx’s drug.
Amylyx did not immediately disclose the price on Thursday, but said it planned to announce the information in a call with investors on Friday morning. In Canada, where the drug received approval in June, the company has proposed a price equivalent to $165,000.
An outside group looking at the cost-effectiveness of new treatments pegged the drug’s value at between $9,100 and $30,700 per year. The Institute for Clinical and Economic Review also noted that many patients are expected to take the Amylyx drug in combination with an older therapy, which costs more than $170,000 a year.
Sunny Brous was diagnosed with ALS in 2015 and hopes to add Relyvrio to the two older FDA-approved medications she already takes for the disease.
“I’ve come this far without this drug and I hope having it now will extend my life further,” said Brous, 35, who lives near Fort Worth, Texas.
The Amylyx data comes from a study in 137 patients that showed some benefit in slowing the disease, based on functionality questionnaires completed by the patients. Patients who continued to take Relyvrio after the study ended appeared to survive several months longer than patients who originally received a placebo, the FDA noted.
“The ALS community has shown that our advocacy can affect the decisions that are made about our health,” said Larry Falivena, an ALS Association board member who was diagnosed with the disease in 2017, in a statement sent by email. email. The group invested $2.2 million in Amylyx’s initial research and expects to recoup $3.3 million from future drug sales.
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