WASHINGTON (AP) — A controversial drug for Lou Gehrig’s disease won U.S. approval on Thursday, a long-sought victory for patients that could rekindle concerns about the scientific rigor behind government scrutiny of experimental drugs sexual questioning.
The U.S. Food and Drug Administration approved Amylyx Pharmaceuticals’ drug based on the results of a small mid-stage study in which patients with the debilitating disease appeared to progress more slowly and survive for several months longer. Typically, the FDA requires two large studies or one study with “very convincing” survival results for approval.
“This approval provides another important treatment option for ALS, a currently incurable, life-threatening disease,” Dr. Billy Dunn, FDA’s Director of Drugs for Neurology, said in a statement.
The drug, which will be marketed as Relyvrio, is the third U.S.-approved treatment for amyotrophic lateral sclerosis, or ALS, that destroys nerve cells needed for basic functions such as walking, speaking and swallowing.
The FDA’s review has become a flashpoint in a wider debate about the regulator, including how much flexibility it should have in reviewing drugs for deadly diseases and how much weight it should give to calls from patients and other outside voices.
“I think it shows the recklessness of the FDA, and I think it shows the tenacity of ALS patients and advocates,” said Dr. Catherine Lomen-Hoerth, an ALS expert at the University of California, San Francisco. “The company is really doing everything possible to get this potential drug to patients.”
Amylyx’s drug is the latest in a string of neurological drugs that have been approved by the FDA despite questionable efficacy data. The agency still faces two government investigations into its approval of the Alzheimer’s drug Aduhelm last year, but it has yet to prove the drug slows the disease.
The latest approval followed a very turbulent path that included two negative comments from scientists inside the FDA who called the company’s results “borderline” and “unconvincing.” An outside advisory panel backed the negative opinion in March, voting narrowly against the drug.
But the FDA faces intense pressure from ALS patients, advocates and members of Congress. In recent weeks, the agency has received more than 1,300 written comments from the ALS community on supportive care.
That outpouring helped shake up the same panel of experts when the FDA earlier reconvened them to revisit Amylyx’s drug. The second time, they supported the drug, 7-2. The vote was not binding, but appeared to open the door for FDA approval.
Several panelists said they were reassured by a special exchange at the meeting in which FDA officials demanded — Amylyx confirmed — that the company would voluntarily withdraw its drug from the market if a large, ongoing study didn’t prove its benefit.
The 600-patient study is expected to report results in 2024.
But experts point to many potential problems with such informal commitments. The FDA and the company may disagree on whether the final data supports the drug; or the company that acquires the drug in the future may not be bound by Amylyx’s promise.
The powder-based drug is a combination of two older ingredients: a prescription drug for liver disease and a dietary supplement related to 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 the two drugs separately. But the FDA approval is expected to force insurers to cover the cost of the treatment, raising questions about its price.
Amylyx did not immediately announce the price on Thursday, but said it plans to release the information on a conference call with investors Friday morning.
An outside group analyzing the cost-effectiveness of new treatments has put the drug at between $9,100 and $30,700 a year. The Institute for Clinical and Economic Review also noted that many patients are expected to use Amylyx’s drug in combination with older treatments, costing more than $170,000 a year.
Sunny Brous was diagnosed with ALS in 2015 and wanted to add Amylyx’s drug to two earlier FDA-approved drugs she was already taking for the disease.
“I’ve done this without the drug, and I hope having it now will extend my life even further,” said Bruce, 35, who lives near Fort Worth, Texas.
Amylyx’s data comes from a study of 137 patients that showed some benefit in slowing the disease based on functional questionnaires completed by patients. The FDA noted that patients who continued to take the drug after the study ended appeared to survive several months longer than those who initially received a placebo.
“The ALS community has shown that our advocacy can influence the perception of our health,” ALS Association board member Larry Falivena, who was diagnosed with the disease in 2017, said in an emailed statement. Decision.” The group invested $2.2 million in Amylyx’s early-stage research and will recoup $3.3 million from future drug sales.
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Follow Matthew Perrone on Twitter: @AP_FDAwriter
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The Associated Press Health and Science Division was supported by the Howard Hughes Medical Institute Division of Science Education. The Associated Press is solely responsible for all content.