Regulatory Flexibility: US FDA Approves Amylyx’s Relyvrio For ALS Despite ‘Degree Of Residual Uncertainty’
Executive Summary
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.
You may also be interested in...
Amylyx’s ALS Drug Relyvrio Fails In PHOENIX Confirmatory Study, Setting Up Withdrawal Question
Company will meet with regulators and the broader ALS community to discuss top-line results and make ‘informed decisions,’ which may include voluntary withdrawal. During FDA review, Amylyx had pledged to remove Relyvrio if PHOENIX failed.
US FDA Versus Its Own Precedents
The US FDA has shown astonishing flexibility in approving new drugs for neurodegenerative diseases based on efficacy evidence that falls well short of historical standards. Now the agency faces the delicate challenge of demonstrating that there are still some lines it will not cross.
Regulatory Flexibility: BrainStorm’s Problematic NurOwn BLA May Be A Bridge Too Far For US FDA
Litany of deficiencies, including product quality shortcomings and a failed Phase III clinical efficacy study, may make it impossible for the agency to exercise the type of flexibility it has with the approval of other treatments for ALS and neurodegenerative diseases.