FDA advisory committee votes to support approval of ALS therapy
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Amylyx Pharmaceuticals announced that the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee voted to support approval of the company’s oral, fixed-dose therapeutic AMX0035 for the treatment of ALS.
According to a release from Amylyx, the committee voted 7-2 in favor, ruling that available evidence of the drug’s effectiveness was sufficient. The ruling body’s decision was based on a review of all available evidence, including new analyses submitted for discussion at the Sept. 7 meeting, as well as information presented at the March 30, 2022, PCNS meeting.
“The committee’s thoughtful review of the data and support of the benefit that AMX0035 may bring to the ALS community, if approved, is promising,” Jamie Timmons, MD, head of scientific communications at Amylyx, said in the release. “The CENTAUR trial data has consistently demonstrated potential benefits of AMX0035 on function and overall survival.”
According to the release, AMX0035 combines sodium phenylbutyrate and taurursodiol and may reduce neuronal cell death by alleviating endoplasmic reticulum stress and mitochondrial dysfunction. The therapeutic was studied in the multicenter, phase 2 CENTAUR clinical trial, which enrolled 137 patients with ALS and included a 6-month randomized, placebo-controlled phase as well as an open-label extension, long-term follow-up phase.
The trial met its primary efficacy endpoint, the company stated, with administration of AMX0035 (plus standard of care) significantly slowing the rate of functional decline measured by the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) total score compared with placebo (plus standard of care) at the end of the 24-week randomized phase.
According to the release, results of the trial also indicated that AMX0035 was generally well-tolerated, with similar rates of adverse events and discontinuations recorded in the AMX0035 and placebo groups during the 24-week randomized phase.
The company stated that the committee’s recommendations are not binding and will be considered by the FDA in its review of the pending new drug application for AMX0035.
The Prescription Drug User Fee Act target action date for the NDA is Sept. 29, which was extended by the FDA to allow more time to review additional analyses of data from the company’s clinical studies, per the release.