US – Aduhelm’s approval has been the subject of extensive misinformation and misunderstanding as experts believed data from the first and second phase were not as exemplary as the third phase trials.

The approval of Aduhelm by the U.S. Food and Drug Administration (FDA) came after an extensive development, testing and review process hence granting Biogen an accelerated approval.

Biogen has stated that it is open to formal review and interactions between the FDA, members of the public and other concerned relevant authorities.

Accelerated approval by the FDA

The FDA instituted its Accelerated Approval Program in 1992 to allow for earlier approval of drugs that treat serious conditions, and to fill an unmet medical need based on a surrogate endpoint that is reasonably likely to predict a clinical outcome.

Since 1992, 253 accelerated approvals have been granted to drugs to treat HIV/AIDS, sickle cell anemia, Duchenne muscular dystrophy (DMD), multiple sclerosis (MS) and particularly in the oncology therapeutic area.

In oncology, for example, the surrogate may be tumor shrinkage, as this is likely to predict increased survival. Many cancer patients have benefitted from novel immunotherapy treatments that have received accelerated approval, and death rates from cancer have declined dramatically.

The accelerated approval of Aduhelm was granted based on data from clinical trials showing the effect of Aduhelm on reducing amyloid beta plaques, a surrogate biomarker that is reasonably likely to predict clinical benefit, in this case a reduction in the rate of clinical decline.

Biogen believe more data will be updated continually as the EMBARK study progresses post-marketing confirmatory trial.

The review process that led to accelerated approval was extensive and thorough, during which we responded to numerous questions and requests from the FDA.

Biogen’s approval is supported by data of more than 3,000 patients and 2.2 million pages of clinical data and analyses.

The primary and secondary endpoints had been pre-specified in the Phase 3 trial protocols, before the first patient was enrolled into the trials.

Aduhelm label shows the results on these pre-specified endpoints, based on data that had already been collected at the sites by the time the trials were prematurely terminated on March 21, 2019.

Safety data were also extensively reviewed and well documented in the label, to enable physicians make informed benefit-risk decisions and take appropriate actions as they monitor their patients under treatment.

 FDA approved the drug based on comprehensively reviewed studies that relayed Aduhelm’s consistency in reducing levels of amyloid plaques in the brain.

The FDA further added that although the data was quite complicated with respect to its clinical benefits, they believe that the substantial evidence that shows Aduhelm effectively reducing amyloid beta plaques was reasonable enough to benefit patients.

FDA further requests Biogen to conduct a post-approval clinical trial to verify the drug’s clinical benefit.

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