The FDA has only approved two other ALS drugs. Riluzolea tablet approved in 1995, can last a few months, and typically costs significantly less than $10,000 a year. Edaravone, marketed as Radicava, can slow the progression of symptoms by about 33%. Radicava, originally approved in 2017 as an intravenous infusion, was approved this year as an oral form with a list price of $171,000 a year. Amylyx officials said they expected that, like in the company’s clinical trials, more patients would take Relyvrio, a powder mixed with water, along with one or both other drugs. .
Relyvrio was formed by Amylyx founders Mr Klee and Joshua Cohen, when they were college students at Brown University less than a decade ago. They suggest that combining taurursodiol, an over-the-counter supplement sometimes used to regulate liver enzymes, and sodium phenylbutyrate, a prescription medication for urea disorders in children, may protect cells. neurons in the brain from damage in diseases like ALS by preventing dysfunction of two structures in the cell: the mitochondria and the endoplasmic reticulum.
In an interview, Mr. Klee said the company expects private insurers to cover the drug with no co-pays for patients, and he said Amylyx will work to make it worthwhile. affordable for people on Medicare or Medicaid and free for those who don’t. The insurance company is having financial difficulties. Klee noted that patients have been buying the ingredients themselves for a while, buying taurursodiol supplements from Amazon and paying up to $11,000 a month for sodium phenylbutyrate.
“Now that our product has been approved, we have to focus on making sure it is accessible to everyone,” Mr. Klee said.
The Relyvrio clinical trials included patients who developed symptoms of ALS within the 18 months prior to the trial and were affected in at least three body regions, which is often a sign of rapidly progressing disease. FDA approval does not limit which patients can use the drug. Mr. Cohen said in an interview that Amylyx has no predictions about the number will.
“There are new patients receiving a diagnosis today, and there are patients making end-of-life decisions today, and I think people in different circumstances will make treatment decisions. very different,” he said.
Amylyx has also conducted a small trial of Relyvrio in Alzheimer’s patients, and Mr. Cohen discussed plans to test it for other neurodevelopmental disorders.
“We envision this drug targeting neuronal death pathways, and neuronal cell death is an important part of not only ALS but potentially many diseases,” he said.