An advisory committee of the FDA voted against the approval of Amylyx Pharmaceuticals Inc's (NASDAQ:AMLX) AMX0035, an investigational therapy for amyotrophic lateral sclerosis.
- On the question of "do the data from single-trial & open-label study establish a conclusion that the drug is effective?" four panelists voted "Yes," while six committee members voted "No."
- The FDA's advisory committees issue non-binding recommendations. However, the regulator usually follows them before finalizing authorizations, with an assigned PDUFA action date of June 29, 2022, for AMX0035.
- Related: Amylyx Shares Plummet On Skeptical FDA Adcomm Docs On ALS Drug.
- Amylyx's application is underpinned by a Phase 2/3 trial called Centaur and an open-label extension, which showed a slowing disease progression.
- But the single study was "not exceptionally persuasive," according to the briefing document the FDA released ahead of the meeting.
- Amylyx is already conducting a Phase 3 trial called Phoenix but is hoping for accelerated approval.
- The company held cash and equivalents of $96.1 million as of December 31, 2021.
- Price Action: Currently, Amylyx shares are on hold for trading.
