A much-controversial drug for Lou Gehrig’s disease won US approval on Thursday, a long-awaited victory for patients that is likely to renew questions about the rigor of science. behind government reviews of experimental drugs.
The Food and Drug Administration approved Amylyx Pharmaceuticals’ drug based on results from a small, mid-term study in which patients with debilitating illnesses appeared to progress more slowly and survived longer months. Typically, the FDA requires two large studies or one study with “very convincing” survival results to be approved.
“This approval provides another important treatment option for ALS, a life-threatening disease for which there is currently no cure,” FDA director of neuropharmaceuticals Dr. Billy Dunn said in a statement. Father.
The drug, Relyvrio, is the third drug approved in the United States for amyotrophic lateral sclerosis, or ALS, which destroys nerve cells needed for basic functions such as walking, speaking and swallowing. About 20,000 people in the US are living with the disease.
The FDA review has become a focal point in broader debates about the regulator, including how flexible the agency is when it comes to drugs to treat deadly diseases and weight levels. agency to appeals from patients and other outside voices.
Dr Catherine Lomen-Hoerth, an ALS expert at the University of California San Francisco, said: “I think it demonstrates the FDA’s ability to take it easy, and I think it shows a lot of patient persistence and those of ALS advocate. “The company has really tried to do everything it can to bring this promising drug to patients.”
Amylyx is the latest in a string of psychoactive drugs to win FDA approval despite questionable efficacy data. The agency is still facing two government investigations into last year’s approval of the Alzheimer’s drug Aduhelm, which has not been shown to slow 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 significant need to unanswered.”
The latest approval follows a remarkably tumultuous road, including two negative reviews by internal FDA scientists, who called the company’s results “boundary” and “inconclusive.” .” A panel of outside advisers backed that negative stance in March, narrowly voting against the drug.
But the FDA has faced great 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 advocating the treatment.
That outburst helped shake up the same panel of experts when the FDA reconvened them earlier this month to review Amylyx’s drug. The second time, they favor the drug, 7-2. The vote is non-binding, but it appears to open the door to FDA approval.
Several panelists said they were also reassured by an unusual exchange at the meeting that FDA’s Dr. Dunn requested – and Amylyx asserted – that the company would voluntarily withdraw its drug from the market if A large ongoing study does not confirm benefits.
That study of 600 patients is expected to report results in 2024.
But experts have pointed to many potential problems with such an informal commitment. FDA and company may disagree on whether the data ultimately supports the drug; or a company acquiring 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 liver disorder medicine and a functional food combined with traditional Chinese medicine. Cambridge, Massachusetts-based Amylyx patented the combination and say the chemicals work together to protect cells from premature death.
Some ALS patients took both drugs separately, sometimes paying $5,000 a month for the prescription ingredient, according to doctors. FDA approval is expected to force insurance companies to cover Amylyx’s drug.
Amylyx did not immediately disclose pricing on Thursday but said it plans to release the information during a call with investors on Friday morning. In Canada, where the drug received approval in June, the company proposed a price equivalent to $165,000.
An outside team analyzing the cost-effectiveness of new treatments pegged the value of the drug to between $9,100 and $30,700 per year. The Institute for Clinical and Economic Research also notes that many patients are expected to take Amylyx’s drug in combination with an older therapy, which costs more than $170,000 per year.
Sunny Brous was diagnosed with ALS in 2015 and hopes to be able to add Relyvrio to two older FDA-approved drugs she already takes to treat the condition.
“I’ve come this far without this drug and I hope having it now extends my life even further,” said Brous, 35, who lives near Fort Worth, Texas.
Amylyx data from a 6-month study of 137 patients showed some benefit in slowing disease, based on functional questionnaires that patients completed. Patients who continued to take Relyvrio after the study concluded appeared to live longer than patients who were initially given a placebo, the FDA noted.
Larry Falivena, ALS board member who was diagnosed with the condition in 2017, said: “The ALS community has proven that our advocacy can impact decisions that are being made about health. our health. The team invested $2.2 million in Amylyx’s initial research and was likely to recoup $3.3 million from the sale of the drug.
