The FDA has approved only two other drugs for ALS. Riluzole, a tablet approved in 1995, can extend survival by several months and typically costs much less than $10,000 a year. Edaravone, marketed as Radicava, can slow the progression of symptoms by about 33 percent. Radicava, which was originally approved in 2017 as an intravenous infusion, was approved this year in an oral form that has a list price of $171,000 a year. Amylyx officials said they expected that, as in the company’s clinical trials, many patients would take Relyvrio, a powder that is mixed with water, along with one or both of the other drugs.
Relyvrio was conceived by Amylyx founders Mr. Klee and Joshua Cohen when they were undergraduates at Brown University less than a decade ago. They proposed that combining taurursodiol, an over-the-counter supplement sometimes used to regulate liver enzymes, and sodium phenylbutyrate, a drug prescribed for a pediatric urea disorder, might protect neurons in the brain from damage in diseases such as ALS by preventing the dysfunction of two structures in cells: mitochondria and endoplasmic reticulum.
In an interview, Mr. Klee said the company expected private insurers to cover the drug with no copays for patients, saying Amylyx would work to make it affordable for people with Medicare or Medicaid and provide it for free to those without. they had it sure they are struggling financially. Mr. Klee noted that patients had been getting the ingredients on their own for some time, buying the taurursodiol supplement from Amazon and paying up to $11,000 a month for sodium phenylbutyrate.
“Now that our product is approved, we have to focus on making sure people can access it,” Klee said.
Clinical trials of Relyvrio included patients who developed ALS symptoms within 18 months prior to the trial and were affected in at least three regions of the body, which is usually a sign of rapidly progressing disease. The FDA approval did not restrict which patients could use the drug. Cohen said in an interview that Amylyx had no projections of how many would do so.
“There are patients who are just being diagnosed today, and there are patients who are making end-of-life decisions today, and I think people in different circumstances are going to make very different treatment decisions,” he said.
Amylyx also conducted a small trial of Relyvrio in Alzheimer’s patients, and Mr. Cohen discussed plans to test it in other neurodevelopmental disorders.
“We designed the drug to try to target neuronal death pathways,” he said, “and neuronal death is an important part of not only ALS, but potentially many diseases.”