The maker of a Lou Gehrig’s disease drug that recently failed in a large trial said Thursday that it will withdraw the medicine from the market, acknowledging that it has not helped patients with the deadly neurological disease.
Amylyx Pharmaceuticals announced it will voluntarily stop selling and marketing the drug in the United States and Canada, where new patients will no longer be able to get a prescription.
“While this is a challenging time for the ALS community, we have achieved this path in collaboration with stakeholders who will be affected and in line with our ongoing commitment to people living with ALS,” executives said of the company in a note. Patients already in therapy who wish to continue it will be able to enroll in a program to receive it free of charge.
The Food and Drug Administration approved the much-debated drug Relyvrio in September 2022, following a years-long advocacy campaign by patients with amyotrophic lateral sclerosis, or ALS.
The drug’s failure is a bitter disappointment to patients and advocates, who have lobbied the FDA and other federal agencies to fund and approve more experimental therapies for the deadly muscle-wasting disease.
Relyvrio’s withdrawal leaves just three ALS drugs available to U.S. patients, only one of which has been shown to extend survival by several months.
Amylyx, based in Cambridge, Massachusetts, said in March it was considering withdrawing its drug after a 600-patient clinical trial failed to show any improvement in survival or other health metrics, such as muscle strength or the ability to walk.
The company’s voluntary action resolves what could have been a major dilemma for the FDA. The agency’s regulators would have had no clear path to quickly remove the drug from the market if the company refused to remove it. That’s because the FDA granted the drug full approval, despite the preliminary nature of the company’s efficacy data.
The 2022 approval was based primarily on the results of a small mid-stage study that was criticized by some of the agency’s internal scientists. The agency normally requires two large, late-stage studies showing a clear benefit before granting approval. But FDA officials explained at the time that “regulatory flexibility” was appropriate when reviewing Relyvrio, “given the serious and life-threatening nature of ALS and the substantial unmet need.”
The drug is among a series of drugs for life-threatening and degenerative diseases that have won FDA approval in recent years despite questionable evidence that they work.
ALS gradually destroys nerve cells and the connections needed to walk, talk, talk and breathe. Most patients die within three to five years of diagnosis.
Relyvrio is a powder that combines two older medications: a prescription drug for liver disorders and a dietary supplement associated with traditional Chinese medicine.
Amylyx has been criticized for pricing the drug at $158,000 for a year’s supply. Sales have been disappointing, with some patients stopping the medicine after only a few months.