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Two years ago, I wrote an article here about a new drug that was awaiting approval for ALS patients (who sadly have few treatment options).This drug (Relyvrio, a mixture of sodium phenylbutyrate and taurodiol) has been approved Its developer (Amylyx) conducted a phase 2 trial, but as you can see from the previous post and the references therein, the data was not convincing. The trial had numerous statistical problems, and even so, it did little to prove efficacy. When the FDA’s own statisticians tried to correct the gaps in the data, it seemed to make the readings worse each time. A ruthless point of view is Amylyx (NASDAQ: AMLX) finally presents the clinical protocol from the perspective that only made it possible Believing the drug was effective, an FDA advisory committee ultimately voted against approval.
But as you will see this timeline, the story is not over yet. The second advisory committee meeting was held, and if that sounds strange to you, it should be. This is a very unusual thing. This vote was positive, and the FDA did approve the drug in September 2022. There’s one condition, though: Amylyx is already in a Phase III trial, and if that trial shows it’s ineffective, they’ve promised to withdraw the drug.This medicine has been Sell Excellent, higher than investors expected.in a paper Published in December, a post hoc analysis of phase II (137 patients) showed reductions in inflammatory biomarkers. so…
You know what happens next. Phase III trial just read out, and Relyvrio shows no signs of actually working. Not for the primary endpoint (improvement on the ALS Functional Assessment Scale compared with placebo), nor for any of the secondary endpoints (patient self-report, respiratory function, overall survival, etc.). This medication does not work and will never work. Did. Fortunately, it does have very good safety features, so it seems unlikely that anyone has been physically harmed over the past year and a half. But ALS patients (or their insurance companies) have the opportunity to pay for one drug (listed at $158,000 per year, who knows the actual price is less than that) and ultimately a combination of two generic drugs. They might as well get mints because it has so many benefits. Amylyx says they will announce plans for the drug in the coming weeks, but what might those plans be? To which landfill?
As with the recent Alzheimer’s approval, patient advocacy groups scored a victory following Relyvrio’s approval, and they were a large part of the pressure to force the FDA to reverse its initially correct decision. Congratulations?I hate this, but I still believe it Unmet medical need itself is not There are good reasons to approve the drug. You need what you’ve always wanted: efficacy and safety. Boring, boring, expensive efficacy and safety. Desire and despair don’t matter – in a completely different universe, one where the laws of physics respect and validate human emotions and human pain, they would emerge. But we don’t live in that world. We never have.
Postscript: There is also a surprising development in today’s news—— FDA has announced They will convene an advisory panel for Eli Lilly’s (LLY) new Alzheimer’s disease antibody donanezumab. Many expected it to be approved soon, so this was quite unexpected. But unless the FDA decides to change its mind (or changes its mind, depending on how you look at it), they’re going to approve it no matter what the advisory committee says. Isn’t this the same in modern society?
Reveal: not any
Editor’s note: Summary highlights for this article were selected by Seeking Alpha editors.
