The maker of the newest treatment approved for amyotrophic lateral sclerosis said Thursday it will pull the drug from the market because a large clinical trial did not prove the treatment worked.
Amylyx Pharmaceuticals said in a statement that it has begun the process of withdrawing the drug in the United States, where it is called Relyvrio, and in Canada, where it is called Albrioza. As of Thursday, no new patients will be able to start the drug, while current patients who wish to continue taking the drug can switch to a free drug plan, the company said.
The drug is one of only a few treatments for the severe 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 go-ahead anyway, rather than wait two years for the results of a large clinical trial, citing data showing the treatment is safe and the desperation of ALS patients. The disease robs patients of their ability to control muscles, speak and breathe unaided and often results in 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 either drunk or swallowed through a feeding tube. His list price was $158,000 a year.
Last month, Amylyx, of Cambridge, Massachusetts, announced that the results of a 48-week trial in 664 patients showed that the treatment worked no better than a placebo. The company said at the time that it would consider withdrawing the drug from the market.
On Thursday, Justin Klee and Joshua Cohen, CEOs of Amylyx, said: “While this is a difficult time for the ALS community, we have arrived at this path forward in collaboration with the stakeholders who will be affected and in line with our steadfast commitment to people living with ALS and other neurodegenerative diseases.”
The company said it would cut its workforce by 70%. He also said he is continuing to study the drug in two rare diseases, Wolfram syndrome and progressive supranuclear palsy.
This week, a law firm announced it had filed a class-action lawsuit against Amylyx on behalf of investors who bought the company’s stock. The lawsuit alleges that Amylyx overstated the commercial prospects of Relyvrio, 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 tried to hide negative information from investors by preventing analysts from seeing prescription data for Relyvrio.
Mr. Klee and Mr. Cohen conceived Relyvrio about a decade ago as undergraduates at Brown University. Their idea was that the combination of taurosodiol, a supplement sometimes used to regulate liver enzymes, and sodium phenylbutyrate, a drug for a pediatric uric acid disorder, could protect neurons in the brain from damage in diseases such as ALS, by preventing two structures in cells from malfunctioning: mitochondria. and the endoplasmic reticulum.
The FDA usually requires two conclusive clinical trials, usually 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 came only from a Phase 2 trial in which 137 patients received either the drug or a placebo, as well as an extension study that followed some patients after the trial ended while 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 is completed in 2024. ALS advocacy groups campaigned hard to get the FDA to reconsider.
In March 2022, a panel of independent FDA advisors decided by a narrow margin that the treatment had not yet been proven effective, a conclusion that FDA reviewers themselves reached. The agency then allowed Amylyx to submit more data and took the unusual step of scheduling a second independent advisory panel in September 2022. In a report presented there, the agency’s 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 if, if the treatment received approval but later failed a Phase 3 trial, it would voluntarily stop selling the drug.
Mr. Klee responded that if the trial “is not successful, we will do what is right for patients, which includes voluntarily removing the product from the market.”
This commitment, as well as emotional testimony from patients and doctors, convinced seven members of the advisory committee to favor approval, with only two opposed. Later that month, the FDA granted approval, writing that there was “remaining uncertainty about the efficacy evidence” but that “given the severe and life-threatening nature of ALS and the substantial unmet need, this level of uncertainty is acceptable in this case.”
