Congressional report faults FDA and Biogen for Alzheimer’s drug approval, rollout

Aduhelm, Biogen's controversial drug for early Alzheimer's disease, is seen at Butler Hospital, one of the clinical research sites, on June 16, 2021.Jessica Rinaldi/Globe Staff

An 18-month congressional investigation found significant problems with the approval process the Food and Drug Administration undertook with an Alzheimer’s drug developed by Biogen, calling it “highly atypical” and deviating from “guidance and procedures in significant respects,” according to a report released Thursday.

The report by two congressional committees also faulted the Cambridge-based drugmaker for its “aggressive launch plans” and pricing of the drug despite concerns over the “efficacy, safety, and affordability” of the treatment.

Congressional investigators found that Biogen anticipated spending more than $3.3 billion on sales and marketing of the drug, called Aduhelm, from 2020 to 2024 — more than 2 1/2 times what Biogen spent developing the medicine from 2007 until its approval in 2021.

The report criticized Biogen for setting the price of the drug at $56,000 a year, “despite a lack of demonstrated clinical benefit in a broad patient population.” Medicare — which the report said Biogen knew would shoulder most of the expense of the drug — dramatically limited access to the drug earlier this year.

Investigators concluded that the FDA’s process for approving Aduhelm was “rife with irregularities,’' including dozens of undocumented or poorly documented meetings between agency regulators and company representatives before the approval.

In a statement, the FDA said that it “fully cooperated with the Committees’ evaluation and we continue to review their findings and recommendations.”

The agency said that it conducted an internal review and concluded its employees’ interactions with Biogen representatives “were appropriate.” Nonetheless, the FDA said it had started to make changes consistent with recommendations made by congressional investigators to restore public trust in drug approvals.

Biogen, for its part, cited the FDA’s internal review and said in a statement that the company “stands by the integrity of the actions we have taken.”

The FDA approved Aduhelm in June 2021, making it the first new Alzheimer’s treatment since 2003. The authorization came over the objections of an independent panel of scientific advisers who cited scant evidence that the drug works and concerns about serious potential side effects. Three members of the panel quit in protest after the approval.

On Thursday, one of the members who resigned, Dr. Aaron Kesselheim, a Harvard professor of medicine who had called Aduhelm’s approval “perhaps the worst” drug authorization in the agency’s recent history, said he hadn’t finished reading the congressional report yet but was heartened by it.

“I think it validates a lot of the concerns that other members of the Advisory Committee and I had about the very strange way that meeting was conducted and about the problematic process that FDA went through to give the drug accelerated approval despite it lacking reasonable evidence that worked and its important side effects,” he said.

“The FDA is the most important public health regulator in the US and, I believe, the world, but from what I’ve read it sounds like congressional investigators uncovered numerous instances of undisclosed meetings with the company and internal disagreement being sidelined, which is very troubling,” he added.

Biogen is currently awaiting FDA approval for a new Alzheimer’s drug made with its Japanese partner, Eisai. Lecanemab, use of which led to “moderately less” cognitive decline in a late-stage clinical trial, could be approved as early as January.

This is a developing story and will be updated.