Big reversal: Amylyx ALS drug approved by Adcomm 7:2
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Big reversal: Amylyx ALS drug approved by Adcomm 7:2
Will the FDA follow Adcomm’s advice as usual?
The FDA advisory committee met again to discuss Amylyx’s controversial amyotrophic lateral sclerosis (ALS) drug, Albrioza (AMX0035).
Previously, at a meeting in March, the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee voted 6:4 to express doubts about the persuasiveness of the Phase II data — including the small size of the trial, missing data, the use of analysis of primary Appropriateness of statistical methods for endpoints and issues of randomization.
These ultimately point to the conclusion that the Phase II results, while statistically significant, are not sufficient to confirm that AMX0035 can improve disease progression.
Amylyx put some hope in the second meeting after a briefing from the FDA foretold what appeared to be a failed outcome — Amylyx’s new analysis still wasn’t “sufficiently independent or convincing” to support AMX0035’s effectiveness.
However, the results of the meeting, announced on September 8, achieved a major reversal — external experts on the FDA advisory committee voted 7-2 in favor of approval of AMX0035.
More unusually, CDER’s director of neuroscience, Billy Dunn, and Amylyx’s co-CEO, both pledged to withdraw AMX0035 from the market if it failed the ongoing Phase III trial.
It is reported that in this document update, Amylyx submitted a new analysis of AMX0035 survival data, as well as biomarker data from a Phase II study in Alzheimer’s disease, to show the effect of AMX0035 on neurodegeneration.
One of the new analyses used participants’ response rates to assess treatment effects. However, the FDA has suggested in previous briefings that it is difficult to treat these data as independent confirmatory evidence because they use the same data as the previous analysis.
Amylyx also demonstrated AMX0035’s survival prediction algorithm, created from natural history data from more than 10,000 ALS patients from 12 specialty centers in Europe. However, the FDA rejected the data because the patients in the database were not enrolled in clinical trials, and there were various concerns about the reliability of the analysis, such as external controls or a lack of pre-specified protocols.
In addition, Amylyx also attempted to use a placebo crossover design to estimate the survival benefit of the treatment. The FDA flagged it as not new data, but a new analysis of the same survival data in the original submission, concerned that Amylyx “relies heavily on untested assumptions.” Amylyx does not implement best practices for analysis, including consideration of other methods for sensitivity analysis.
As for the biomarker results from the AD study, the FDA noted that the relevance to ALS is unclear — “even if they prove beneficial in AD, it’s unclear whether they can be generalized to ALS.”
Adcomm’s attitude has taken an unusual turn, perhaps because of the strong demand for treatments in the ALS patient population.
According to Amylyx, the results of the Phase III study of AMX0035 will not be available until 2024. For ALS patients with only 3-5 years of survival, time is life. For example, the American ALS Association previously stated that AMX0035 has been proven to be relatively safe and can be administered orally. For the “terminal disease” of ALS, patients prefer to do something rather than just wait.
It is worth noting that based on the results of the Phase II trial, AMX0035 was conditionally approved in Canada in June this year. However, the FDA must be cautious, and the AD drug Aduhelm, which was partially approved by public opinion, is a lesson from the past.
The PDUFA date for AMX0035 is September 29, will the FDA follow Adcomm’s advice as usual? We can wait and see.
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Big reversal: Amylyx ALS drug approved by Adcomm 7:2
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