The maker of the newest approved treatment for amyotrophic lateral sclerosis said Thursday it was pulling the drug from the market because a large clinical trial produced no evidence that the treatment worked.
The company Amylyx Pharmaceuticals said in a statement that it had begun the process of withdrawing the drug in the United States, where it is called Relyvrio, and in Canada, where it is called Albrioza. Starting Thursday, no new patients will be able to start taking the drug, while current patients who wish to continue taking the drug will be able to move to a free medication program, the company said.
The medication is one of the few treatments for this serious neurological disorder. When the Food and Drug Administration approved it in September 2022, the agency concluded that there was not yet enough evidence that the drug could help patients live longer or slow the progression of the disease.
He decided to give the drug the green light anyway, rather than wait two years for results from a large clinical trial, citing data showing the treatment was safe and the desperation of ALS patients. The disease deprives patients of their ability to control muscles, speak and breathe without assistance and often causes death within two to five years.
Since then, about 4,000 patients in the United States have received the treatment, a powder that is mixed with water and drunk or swallowed through a feeding tube. Its list price was $158,000 a year.
Last month, Amylyx, of Cambridge, Massachusetts, announced that results from a 48-week trial of 664 patients showed that the treatment worked no better than a placebo. The company then said it would consider removing the drug from the market.
On Thursday, Justin Klee and Joshua Cohen, co-CEOs of Amylyx, said in a statement: “While this is a difficult time for the ALS community, we reached this path forward in partnership with the stakeholders who will be affected and in in line with our firm commitment to people living with ALS and other neurodegenerative diseases.”
This week, a law firm announced that it had filed a class-action lawsuit against Amylyx on behalf of investors who purchased shares of the company. The lawsuit alleges that Amylyx exaggerated Relyvrio’s business prospects, failed to disclose that patients were stopping treatment after six months and that the rate of new prescriptions was declining. The lawsuit also alleges that the company attempted to hide negative information from investors by preventing analysts from seeing Relyvrio’s prescription data.
Klee and Cohen conceived Relyvrio about a decade ago as undergraduates at Brown University. Their idea was that the combination of taurursodiol, a supplement sometimes used to regulate liver enzymes, and sodium phenylbutyrate, a medication for a pediatric urea disorder, could protect brain neurons from damage in diseases such as ALS by prevent the dysfunction of two structures in cells: the mitochondria. and the endoplasmic reticulum.
The FDA typically requires two compelling clinical trials, typically Phase 3 trials, which are larger and more extensive than Phase 2 studies. For serious diseases with few treatments, the agency may accept one trial plus additional confirmatory data. For Relyvrio, the data comes only from a Phase 2 trial in which 137 patients took the drug or a placebo, plus an extension study that followed some patients after the trial ended when they were knowingly taking the drug.
The agency initially recommended that the company not apply for approval of the drug until the Phase 3 trial was completed in 2024. ALS advocacy groups campaigned vehemently to persuade the FDA to reconsider its decision.
In March 2022, an independent FDA advisory committee narrowly decided that the treatment had not yet been proven effective, a conclusion also reached by the FDA’s own reviewers. The agency then allowed Amylyx to submit more data and took the unusual step of scheduling a second independent advisory committee meeting in September 2022. In a report filed there, agency reviewers said they also found the new data insufficient. .
At that hearing, Dr. Billy Dunn, then director of the FDA’s office of neuroscience, asked the company whether, if the treatment received approval but then failed in the Phase 3 trial, it would voluntarily stop selling the drug.
Klee responded that if the trial “is not successful, we will do what is right for patients, including voluntarily withdrawing the product from the market.”
That commitment, plus emotional testimony from patients and doctors, persuaded seven members of the advisory committee to favor approval, with only two opposed. Later that month, the FDA granted approval, writing that there was “residual uncertainty about the evidence of efficacy,” but that “given the serious and life-threatening nature of ALS and the significant unmet need, this level of uncertainty is acceptable.” In this instance”.
