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The FDA panel voted 7 to 2 in favor of the oral drug, AMX0035, citing the unmet need for more treatments against the fatal neurodegenerative condition commonly known as Lou Gehrig’s disease.
The positive vote comes about five months after the panel declined to recommend FDA approval for AMX0035, citing the lack of “substantially persuasive” data from a mid-stage trial showing the drug’s effectiveness against ALS.
Since then, the company has provided new analyses estimating that the drug could extend life expectancy by nearly 10 months compared with placebo, along with biomarker data from a study of the treatment in Alzheimer’s disease.
The FDA had deferred its decision based on the additional data and convened a rare second meeting.
Patients and experts have been urging the agency to approve Amylyx’s drug due to its relative safety and the expectation of increased survival. Average life expectancy after diagnosis of ALS is two to five years.
ALS, whose cause is largely unknown, breaks down nerve cells in the brain and spinal cord, impacting physical function and resulting in severe disability and death. It affects more than 31,000 patients in the United States, according to CDC data from 2017.
Dr. Liana Apostolova, one of the voting members, said that the new confirmatory evidence is not “clinically persuasive, but nonetheless is quite reassuring.”
The FDA typically follows the panel’s recommendations, though it is not obligated to. The agency’s decision is expected by Sept. 29.
