Amylyx’s ALS Drug Should Wait For Phase III Results, US FDA Panel Says
Six of 10 advisory committee members say Phase II CENTAUR trial cannot stand on its own to support approval at this time. However, patients and advocates say thousands will die if they have to wait for the Phase III PHOENIX trial to complete in 2024 before the drug becomes widely available.
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Agency considered Amylyx’s plan to submit unblinded efficacy data from the ongoing Phase III trial during the ALS drug's NDA review but ultimately decided against it, citing concerns about study integrity and the small amount of data likely to be available.
Real-World Evidence: Amylyx’s Use Of External Controls To Frame Relyvrio’s Survival Benefit Rife With Problems
The post hoc comparison of an exploratory, long-term survival benefit in the CENTAUR trial to historical controls in two ALS patient databases lacked prespecification and a common treatment protocol, and potential differences in prognostic factors may have confounded the results.
Regulatory Flexibility: US FDA Approves Amylyx’s Relyvrio For ALS Despite ‘Degree Of Residual Uncertainty’
The single positive Phase II CENTAUR study, along with confirmatory evidence of benefit on long-term survival in the open-label extension, demonstrate substantial evidence of effectiveness, the agency says, acknowledging a level of uncertainty that is acceptable in the disease context.