Drug maker pulls failed ALS treatment from market in rare move
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The maker of a failed drug meant to treat amyotrophic lateral sclerosis (ALS) said Thursday it will voluntarily pull the treatment from the market, a rare move for a pharmaceutical company. It comes after a clinical trial showed the medication doesn’t help patients with the deadly neurological condition, also known as Lou Gehrig’s disease.
The drug was already controversial, having been approved by the U.S. Food and Drug Administration in 2022 after one small clinical trial while the approval process for new treatments usually requires two large studies that show a clear benefit. That was because FDA officials wanted to give the drug, called Relyvrio, more regulatory flexibility, “given the serious and life-threatening nature of ALS and the substantial unmet need.”
“While this is a difficult moment for the ALS community, we reached this path forward in partnership with the stakeholders who will be impacted and in line with our steadfast commitment to people living with ALS and other neurodegenerative diseases,” the CEOs of the drug maker, Amylyx Pharmaceuticals, said in a statement.
SIGNALS
The FDA has let standards slip, critics say
The FDA has tried to make its rules more flexible in the case of drugs that could help with particularly devastating conditions such as ALS that have few treatment options, but critics say the agency is now giving too much leeway, ignoring its own experts, and setting a bad precedent for future regulations.
“When they lower the standards for one product, however urgently needed it might be, it has a tendency to serve as a model,” the president of the nonprofit National Center for Health Research told The Washington Post in March. Regulatory experts warned Politico that the FDA’s flexibility could be an issue in the future if drug companies don’t voluntarily cease production.
Pledge to remove drug from market if it failed could be a model for others
Those who advocate for more flexibility in the FDA’s regulatory process say this series of events was actually a win for patients. The FDA greenlit the drug in part because Amylyx Pharmaceuticals pledged to pull it from the market if it didn’t prove effective, experts told Politico, and the company kept its word. “This might be an example of successful regulatory flexibility, even though the drug didn’t pan out,” one said.
The ALS Association lauded the company’s handling of the situation. “We believe the example of Relyvrio shows how the system can work,” the group said in a statement. “Safe and potentially effective treatments can be made accessible rapidly until further research can confirm their efficacy.”
Company voluntarily pulling drug prevented major FDA headache
The FDA might have faced a “major dilemma” if Amylyx Pharmaceuticals had not voluntarily pulled the treatment, The Associated Press reported, because the regulatory agency would not have had a quick way to force the drug from the market.
One member of the FDA’s expert panel warned in 2022 stressed how difficult it would be to order the company to stop selling the drug once it was granted full approval, saying “Frankly, I’m not sure it’s ever taken place.”
