Manufacturer to pull controversial ALS drug off the market after failed clinical trial

The maker of a drug to treat Lou Gehrig’s disease (ALS) has announced that it will be withdrawn from the market after a recent failure in a large-scale clinical trial.

Amyryx Pharmaceuticals announced that it will cease sales and marketing of Relibri in the United States and Canada and that Relibri will no longer be available to new patients as of Thursday.

“These are difficult times for the ALS community, but we are working with affected stakeholders to reach this path forward, consistent with our firm commitment to people living with ALS and other neurodegenerative diseases. ” said Joshua Cohen, co-CEO of Amirix. Justin Clay said in a statement.

The move comes after the company announced last month that a clinical trial of more than 650 people found that Relibrio was not more effective than a placebo in slowing the progression of ALS and showed no benefit in patients. It was done. The drug also failed to show improvements in the trial’s secondary endpoints, such as muscle strength.

Patients who are already receiving the drug and wish to continue can enroll in a program to receive it free of charge, the company said.

The withdrawal is a blow to ALS patients, who have few treatments available for the progressively debilitating disease. Once developed, ALS progresses, and most people with the disease eventually lose the ability to walk, dress, write, speak, swallow, and breathe. The average survival time is three years, but some people live longer.

The Food and Drug Administration (FDA) approved Relibrio in 2022 based on data from a small trial that showed it modestly slowed the progression of ALS. The approval was controversial, with an outside expert advisory panel initially voting against the drug, expressing concerns about the data from such a small trial.

However, the committee supported the drug after hearing from patients and ALS advocates at a subsequent hearing. The FDA ultimately approved the drug, saying that while there was uncertainty about its effectiveness, it was determined to be acceptable given the pressing need to treat ALS.

The agency typically requires two large Phase 3 trials before deciding on approval, with each trial typically enrolling anywhere from a few hundred people to about 3,000 people. Relyvrio’s data comes from one of his Phase 2 trials, in which 137 patients took the drug or a placebo.

Although the company is already conducting large-scale clinical trials and would no longer need to do so once the drug is fully approved by the FDA, CEOs said they would not move forward if further trials do not show efficacy. He publicly promised to stop taking the drug.

When Relyvrio first hit the market, Amylyx faced criticism for charging nearly $160,000. Only about 4,000 patients in the U.S. have received the treatment since it was approved.