The maker of the latest treatment approved for amyotrophic lateral sclerosis announced Thursday that it was withdrawing the drug from the market after a large-scale clinical trial failed to provide evidence that the treatment is effective. did.
The company, Amyryx Pharmaceuticals, said in a statement that it has begun the process of discontinuing the drug, known as Relibri in the U.S. and Albriosa in Canada. As of Thursday, new patients cannot start receiving the drug, but current patients who want to continue taking the drug can move into the free drug program, the company said.
This drug therapy is one of the few treatments for severe neurological disorders. When the Food and Drug Administration approved the drug in September 2022, it concluded that there was still not enough evidence that the drug would help patients live longer or slow the progression of the disease. I attached it.
Rather than wait two years for results from large-scale clinical trials, the company decided to approve the treatment anyway, citing data showing the treatment was safe and the desperation of ALS patients. The disease robs patients of their ability to control their muscles, speak and breathe without assistance, and often leads to death within two to five years.
Since then, about 4,000 patients in the U.S. have received the treatment, which consists of a powder mixed with water or taken through a feeding tube. List price was $158,000 per year.
Last month, Cambridge, Mass.-based Amyryx announced that a 48-week trial of 664 patients showed the treatment was no more effective than a placebo. The company subsequently announced that it would consider withdrawing the drug from the market.
On Thursday, Amirix Co-Chief Executive Officers Justin Klee and Joshua Cohen said in a statement: This aligns with our strong commitment to people living with ALS and other neurodegenerative diseases. ”
The company announced it would cut its workforce by 70%. The company also said it 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 Amirix on behalf of investors who bought the company's stock. The lawsuit alleges that Amyrix overstated Relibrio's commercial prospects and failed to disclose that patients were discontinuing treatment after six months and that new prescription rates were decreasing. The complaint also alleges that the company tried to hide negative information from investors by preventing analysts from viewing Relibrio prescription data.
Klee and Cohen invented Relibrio about 10 years ago as undergraduates at Brown University. Their idea was to combine taur ursodiol, a supplement sometimes used to regulate liver enzymes, and sodium phenylbutyrate, a drug used to treat childhood urea disease, to improve the ability of mitochondria, two structures within cells. By preventing their dysfunction, the brain's neurons could be protected from damage caused by diseases such as ALS. and the endoplasmic reticulum.
The FDA typically requires two convincing clinical trials, typically a Phase 3 trial that is larger and more extensive than a Phase 2 trial. For serious diseases for which there are few treatments, government agencies can accept his one trial and additional confirmatory data. In the case of Relibrio, the data comes from only one Phase 2 trial in which 137 patients took either the drug or a placebo, and an extension study that followed up some patients who intentionally took the drug after the trial ended. This is what was obtained.
The agency initially recommended not applying for approval until Phase 3 trials are completed in 2024. ALS advocacy groups campaigned heavily to convince the FDA to reconsider.
In March 2022, the FDA's panel of independent advisors decided by a narrow margin that the treatment's effectiveness has not yet been proven, a conclusion reached by the FDA's own reviewers. The agency then took the unusual step of allowing Amirix to submit more data and scheduling a second independent advisory committee meeting for September 2022. In their report, the agency's reviewers also said they believed the new data was insufficient.
At that hearing, Dr. Billy Dunn, then head of the FDA's Office of Neuroscience, said that if a treatment is approved but later fails a Phase 3 trial, the drug will be voluntarily withdrawn from sale. I asked the company whether they would do so.
Klee responded that if the trial was not successful, he would do what was right for patients, including voluntarily removing the product from the market.
This commitment, along with emotional testimony from patients and physicians, convinced seven advisory committee members to vote in favor of approval, with only two opposed. Later that month, the FDA granted approval, saying that while “uncertainty remains regarding the evidence of efficacy,” “Given the serious and life-threatening nature of ALS and the substantial unmet need,” , this level of uncertainty is acceptable in the United States.” this instance. ”