Aducanumab Accelerated Approval Reflects US FDA Flexibility But Raises Doubts About Confirmatory Trial
Biogen/Eisai Alzheimer’s drug wins agency nod on basis of amyloid plaque reduction, a surprising use of the accelerated approval pathway that was not considered by an FDA advisory committee; approval raises feasibility questions about confirmatory trial and daunting prospect of market withdrawal if studies fail to verify clinical benefit.
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Having a confirmatory trial underway at the time of approval should be the ‘default expectation’ and the agency should publicly explain when and why it grants exceptions to this rule, experts said at a National Academies meeting on accelerated approval.
Even before a new law granted FDA authority to require studies be underway before accelerated approval, review divisions have, in some cases, given sponsors years of advance notice on expectations around study timing, according to a Pink Sheet review of NME accelerated approvals.
Although Aduhelm’s accelerated approval in June 2021 ‘generated a significant amount of discussion among external stakeholders,’ CDER’s work on that application ‘led to a leap in our understanding of amyloid as a surrogate marker and a greater understanding of the role that a reduction in amyloid plaque may play in a slowing of the disease,’ drug center director says.