By MATTHEW PERRONE (AP Health Writer)
The company that made a drug for Lou Gehrig’s disease, which failed in a big study, announced it will remove the medicine from the market because it did not help patients with the deadly neurological condition.
Amylyx Pharmaceuticals declared its decision to stop sales and marketing of the drug in the U.S. and Canada voluntarily, making it unavailable for new patients to get a prescription.
The company co-founders said in a statement that although it's a tough time for the ALS community, they made this decision in partnership with impacted stakeholders and in line with their commitment to people living with ALS. Existing patients on the therapy who want to continue can enroll in a program to receive it for free.
The controversial drug, Relyvrio, was approved by the FDA in September 2022, following advocacy by ALS patients.
The drug's failure is a disappointing outcome for patients and advocates who have been urging the FDA and other federal agencies to fund and approve more experimental therapies for the fatal muscle-wasting disease.
The withdrawal of Relyvrio leaves only three ALS medicines available to U.S. patients, with only one of them shown to extend survival by several months.
Amylyx, based in Cambridge, Massachusetts, also announced a major restructuring effort that includes laying off 70% of its more than 350 employees. Company executives plan to continue studying Relyvrio and another experimental drug for several rare diseases, including Wolfram syndrome, which causes childhood diabetes and blindness.
In trading on Thursday morning, the company's shares rose by more than 7.5%.
Last month, Amylyx had mentioned the possibility of pulling its drug after a clinical trial involving 600 patients failed to show any improvements in survival or other health measures, such as muscle strength or walking ability.
The company's voluntary action resolves a potential dilemma for the FDA, which would not have had a clear path to quickly force the drug from the market if the company had refused to remove it. This is because the FDA had granted full approval for the drug, despite the preliminary nature of the company's data on effectiveness.
The approval in 2022 was mostly based on results from a small, mid-stage study, which was criticized by some of the agency's own internal scientists. Normally, the agency requires two large, late-stage studies showing a clear benefit before granting approval. However, regulatory flexibility was deemed appropriate at the time due to the serious and life-threatening nature of ALS and the substantial unmet need.
This medication is part of a series of drugs for deadly, degenerative diseases that have received FDA approval in recent years despite questionable evidence of their effectiveness.
ALS gradually destroys the nerve cells and connections necessary for walking, talking, speaking, and breathing, with most patients succumbing within three to five years of diagnosis.
Relyvrio is a substance made of two old drugs: a prescribed medicine for liver issues and a natural remedy linked to traditional Chinese medicine.
Amylyx received negative feedback for setting the price of the drug at $158,000 for a year’s worth. The sales were not as expected, as some patients stopped using the medicine after just a few months.
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The Associated Press Health and Science Department is supported by the Howard Hughes Medical Institute’s Science and Educational Media Group. The AP is solely responsible for all content.
