Amylyx, adcomm promises and dealing with uncertainty Read in browser Endpoints News Thank you for reading, Brendan Dalton! BASIC SUBSCRIPTION UPGRADE Max Gelman Max Gelman Senior Editor, Endpoints News
What’s the lesson from Amylyx? Amylyx's decision this month to withdraw its ALS drug wrapped up one of the industry’s most fascinating — and sometimes controversial — drug development journeys of the last several years. The company won praise for pulling the drug from the market after a failed Phase 3 trial. But the episode raises more questions than it answers, especially for the FDA. Will this set a precedent for single-trial applications? How will regulatory flexibility be applied going forward, in ALS and other rare neurodegenerative diseases? And when will drug companies produce effective treatments that actually work to treat this devastating and universally fatal disease? The science question may be the most uncertain, but the regulatory one is the most immediate. By approving Amylyx’s drug on limited evidence and then leaving its fate up to the company, the FDA left far too much to chance based on a company’s goodwill, University of Pennsylvania bioethicist Holly Fernandez Lynch told me when we talked earlier this month. (A similar debate about what to do with cancer drugs that don’t end up working is playing out with accelerated approvals, renewed by an April 7 article in JAMA reviewing overall survival results.) What happens when the next biotech with unclear or inadequate evidence acts differently? Just last week, BrainStorm said it will start enrolling a new Phase 3b trial in a last-ditch effort. This comes after it filed for approval last year “over protest” of the FDA’s refusal to accept the company’s application. BrainStorm’s ALS drug has failed a Phase 3 trial and been in a long battle with regulators to keep pushing it, and it ultimately withdrew the application in order to submit a new one in the unlikely event that the additional trial succeeds. Lynch has argued for some kind of middle ground between accelerated and full approval, where drugs that don’t have biomarker data (like Relyvrio) can still be greenlit on a single study. Formalizing this would benefit drugmakers and patients alike — companies can count on flexibility where its needed and patients can get drugs scientifically proven to work. Without such a framework, the agency will by default keep ceding power over these types of decisions.
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