At different times, the US Food and Drug Administration's approval of the drug aducanumab would have been celebrated as one of the great therapeutic advances of 2021. After all, it is the first drug to target the causes of Alzheimer's, the most common form of dementia. .
THEAlzheimer it affects millions of people in the world: 1,4 million in Italy alone. There is a handful of medications that temporarily dull his symptoms, but none could really slow his advance.
Aducanumab, uphill start
Despite the premises, the FDA approval has become one of the most controversial. It all starts in March 2019, when the developers of aducanumab, the American Biogen and the Japanese Eisai, stop two large clinical trials after an independent monitoring committee determines that the drug shows no clinical benefit.
Yet the drug seemed to be doing its job: ridding patients' brains of amyloid plaques, the abnormal protein deposits characteristic of Alzheimer's. However, as it is still unclear whether or not amyloid causes the disease, the matter has been put on hold. However, Biogen did not give up: after further analysis, it resubmitted its case in November 2020.
Another resounding rejection, with 10 out of 11 votes against: "there is no substantial evidence of the effect of aducanumab," the biostatistic office of the FDA said. The risks presented by aducanumab seemed to outweigh the benefits.
End of games for aducanumab? No.
The FDA could have concluded things there. Instead, the agency gave the green light to aducanumab last June, deeming it "reasonably likely to anticipate clinical benefit." Biogen is required to set up another study, with results expected by 2030: in the meantime, it can sell its product (under the trade name Aduhelm) to any patient with Alzheimer's.
On the same day, the company announced the drug's list price in the US: over 50.000 euros a year.
And now it's chaos
Patient associations and researchers have cheered the FDA's decision, hoping that the Biogen drug will work. Three members of the supervisory board's executive board resigned in protest, and it seems like just the beginning.
Even the US Congress has launched two investigations by as many commissions as to how Aducanumab was approved and why it costs so much.
A report emerged (in the press) denouncing a "pressure" campaign initiated by Biogen to influence the FDA.
Another investigation, this time requested by the FDA commissioner himself, Janet Woodcock. Meanwhile, some medical centers say they will not prescribe the drug.
Will it work?
By 2030, when there are reliable results, will we count more successes or more harm from this drug? In Europe, the EMA rejected the use of the drug, too early to know if it's worth it. In the US, doctors are making their own decisions, generally informing patients.
"When I talk to patients about aducanumab, I include a narrative of the events that led to its approval," he says. Jason Karlawish, a geriatrician at the University of Pennsylvania's Perelman School of Medicine.
"People need to understand where something comes from."
