FDA Approves Controversial Drug for Altzheimer's

For the first time in over a decade, the US Food and Drug Administration has approved a new drug for the treatment of Alzheimer's disease, and done so under its accelerated approval process. Alzheimer's disease is the sixth leading cause of death in the US. It is a neurodegenerative disease leading to a progressive and irreversible destruction of neurons resulting in loss of cognitive function and memory. Over time, patients develop severe dementia, loss of independence, and death. So far, pharmacologic treatment options have been extremely limited, with the few previously approved drugs providing only mild symptomatic improvement, but not modifying disease progression - let alone offering a cure.

The US Food and Drug Administration (FDA) approved aducanumab (Aduhelm) in June of this year on the strength of a third clinical trial that followed on the heels of two previous failed trials. The third trial suggested that Aduhelm may delay to progression of the disease by more than 20% by removing the amyloid plaques that are the hallmark of Altzheimer's from the brain.

The decision was controversial in that no prior study has shown that the removal of the plaques produces any clinical benefit. Objections centered on approval of a drug that might provide minimal benefit to a small number of patients at the risk of significant complications, such as mild to severe Amyloid-related imaging abnormalities (ARIA) (abnormal differences seen in brain images of Altzheimer's Disease associated with amyloid-modifying therapies) leading to accelerated functional decline in a much larger group of patients.

Already known side effects include headache, changes in mental state, confusion, vomiting, nausea, tremor and gait disturbances, decreased level of consciousness, diarrhea, delirium and disorientation. The accelerated approval pathway requires the company (Biogen) to verify clinical benefit in a post-approval trial. If the sponsor cannot verify clinical benefit, FDA may initiate proceedings to withdraw approval of the drug. 

I am sure that will be an enormous comfort to the families of those harmed by the drug in the interim.