Biogen’s Accelerated Approval for its Alzheimer’s Drug: Controversial but Routine
(Thursday, June 11, 2021)
This week FDA approved Biogen’s new drug for Alzheimer’s disease, Aduhelm, despite getting a no-approval recommendation from its Advisory Committee. FDA has been strongly criticized in the media for this decision, but this is not the first time FDA made such decision and certainly not the last. There are several reasons why if FDA had rejected the drug, it would have been equally controversial. This approval was made under the Accelerated Approval pathway which specifically allows for such approvals. Biogen had identified a surrogate marker as the primary endpoint for its clinical trials and only had to demonstrate relief in terms of that biomarker, not any other indicators of the disease. There are valid questions about the Biogen changing the original endpoints and going with the surrogate endpoints later in development, but it was done with FDA’s concurrence. The primary endpoint of the clinical trials was met, although its clinical significance was not clear. FDA’s decision was based on the primary endpoint consideration alone, which is legally unchallengeable although controversial. FDA does this routinely in cancer drugs where improvement in surrogate markers is used to approve new drugs with scant evidence for clinical benefits. Basically, for disease with no available treatment, the bar for approval is lowered. Some treatment is better than no treatment. The main controversy seems to be for the financial windfall for Biogen. This is the first treatment of Alzheimer’s disease in 20 years, and Biogen is expected to make tens of billions of dollars from its sales. While for cancer therapies long-term treatments are uncommon, Alzheimer’s patients can survive for years and will need the new drug for the rest of their lives. Similarly controversial were the approval decisions for Sarepta’s Duchenne Muscular Dystrophy drug, or Sprout’s female libido drug before that. In those cases as well, the media criticism was primarily due to the financial benefits to the developers not based on regulatory misconduct by FDA. Biogen, Sarepta and Sprout, all played by the rules and got the better end of the deal. Does not make it unethical or improper. This is a routine decision by the FDA, one that will be repeated, controversy or none.
Dr. Mukesh Kumar
Founder & CEO, FDAMap
Linkedin: Mukesh Kumar, PhD, RAC