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# Amylyx Lou Gehrig’s Disease Drug Approved
- URL: https://www.fdaweb.com/amylyx-lou-gehrigs-disease-drug-approved/
- Published: 2022-09-29T12:00:00.000Z
- Updated: 2026-09-14T18:01:10.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5152862

FDA has approved Amylyx’s amyotrophic lateral sclerosis (ALS; also known as Lou Gehrig's disease) drug AMX0035 (sodium phenylbutyrate and taurursodiol). The agency green light comes after an unprecedented second advisory committee vote within six months reversed its earlier negative recommendation to back the drug in a 7 to 2 vote ([see earlier story](https://www.fdaweb.com/2nd-time-a-charm-in-als-drug-panel-vote/)).

During the panel meeting, the FDA advisors were persuaded by new analyses submitted by the company and by patient pleas for a vote reversal. The company said its new analyses showed the median survival benefit at 9.7 months for patients taking AMX0035\. Patient advocates have pointed to the unmet need for more therapies to treat the fatal neurodegenerative disease. In a briefing document released in advance of the meeting ([see earlier story](https://www.fdaweb.com/data-not-conclusive-on-amylyx-als-drug-fda/)), FDA reviewers appeared pessimistic that new analyses and data from Amylyx would be sufficient to warrant approval. Their [briefing document](https://www.fda.gov/media/161378/download?ref=fdaweb.com) had FDA leaning on its earlier recommendation for a confirmatory trial, made before Amylyx originally submitted the NDA. The agency noted that the company has an ongoing Phase 3 study in 600 patients with ALS worldwide. It has currently enrolled over 50% of the proposed study population and it is expected to complete it in late 2023 to early 2024.

Additionally, FDA has been provided assurance from the company that if the ALS drug were approved and the confirmatory trial failed to show a benefit, then the company would remove it from the market.