FDA Policy/General

2nd Time a Charm in ALS Drug Panel Vote

For the second time in six months, an FDA advisory committee convened to discuss data on Amylyx’s amyotrophic lateral sclerosis (ALS; also known as Lou Gehrig's disease) drug AMX0035 (sodium phenylbutyrate and taurursodiol), but unlike the 3/30 near mixed vote that recommended against approving the therapy, the 9/7 panel vote (7 to 2 in favor of the therapy) gave the company and desperate patients hope that approval could be in their near future. FDA likely will make a final decision by the user fee review action target date of 9/29.

FDA Peripheral and Central Nervous System Drugs Advisory Committee members appeared persuaded by new analyses submitted by the company and patient pleas in their vote reversal. The company says its analyses show 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.

It remains to be seen whether FDA shares in this new backing for the drug. In a briefing document released in advance of the meeting (see earlier story), FDA reviewers appeared pessimistic that new analyses and data from Amylyx were sufficient to warrant approval. The briefing document has FDA leaning on its earlier recommendation for a confirmatory trial, made before Amylyx originally submitted the NDA. The agency notes 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 late 2023 to early 2024.

Additionally, FDA’s briefing document dismissed using the accelerated approval pathway for AMX0035. “Accelerated approval concerns the character of the endpoints, not the strength of the results on those endpoints,” the reviewers wrote. “An effect on an endpoint supporting accelerated approval must be an effect on an endpoint that in its character is reasonably likely to predict clinical benefit and, in its persuasiveness, provides substantial evidence of effectiveness from adequate and well controlled trials, just as substantial evidence of effectiveness on a clinically meaningful endpoint from adequate and well controlled trials supports traditional approval.” They noted that there are no data showing an effect of AMX0035 on an endpoint that is reasonably likely to predict clinical benefit for ALS.

During the panel meeting, FDA was 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.

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome