Biogen, FDA walk back controversial Aduhelm label after weeks of fierce criticism

Biogen biologics plant in Denmark
The FDA and Biogen agreed to narrow the recommended use for the drugmaker's controversial Alzheimer's disease drug Aduhelm. (Biogen)

Following weeks of fiery criticism for its wide-labeled approval for Biogen’s Aduhelm for anyone with Alzheimer’s disease, the FDA is now narrowing the recommended window of patients to only those with milder forms of the memory-robbing disease. 

Biogen on Thursday said the FDA approved an updated label for Aduhelm, also known as aducanumab, that recommends the amyloid-beta targeting antibody for people with mild cognitive impairment or mild dementia, aligned with those included in Biogen’s late-stage trials. 

The FDA warns that there is “no safety or effectiveness data on initiating treatment at earlier or later stages of the disease than were studied.” 

RELATED: Biogen's shockingly broad Aduhelm label—and $56K price—set up a $10B launch, analysts say

Biogen requested the update based on “ongoing conversations with prescribing physicians, FDA and patient advocates,” research head Alfred Sandrock Jr., M.D., Ph.D., said in a statement. The goal is to “further clarify the patient population that was studied across the three Aduhelm clinical trials that supported approval,” Sandrock added.

The slimmed approval, an unusually sudden result given Aduhelm was approved just a month ago, follows the FDA’s initial decision to award the treatment an “almost shockingly broad” label, as Bernstein analyst Ronny Gal put it. The original nod essentially gave Biogen free rein to the estimated 6 million Alzheimer’s patients living in the U.S.

Intense pushback followed as market watchers pointed out that the drug could overwhelm payer budgets, particularly Medicare, which covers most Alzheimer's patients in the U.S. But Biogen has maintained that its launch would only target the 1 to 2 million mild disease patients, rather than storm the entire Alzheimer’s market.

While it might seem that the new label would damage Biogen’s sales prospects, Jefferies analysts said in a note on Thursday that it could help focus reimbursement decisions and deliver Aduhelm to the appropriate patients faster.

The original label “led to negative attention and confusion on who is best suited to get the drug,” and ultimately caused delays while insurers weighed whether they would reimburse everyone, the analysts argued.

RELATED: Congressional Democrats launch probe into Biogen's Aduhelm FDA approval, citing 'serious concerns'

In the meantime, Biogen and the FDA have been left to respond to a string of stinging rebukes. On Capitol Hill, lawmakers have chastised the company's $56,000 per year list price for Aduhelm, the only approved drug intended to slow the progression of the disease.

In some estimations, given the FDA’s expansive go-ahead, Aduhelm could eventually balloon Medicare’s spending above the cost of all current Part B drugs.

That’s all for a drug that has shown murky clinical benefits, at best, critics argue. Both Biogen and the FDA are now the subjects of a Congressional probe, spearheaded by the House Committee on Oversight and Reform, over the company’s pricing tactics, as well as the FDA’s approval process. 

As more details surrounding Aduhelm’s approval come to light, prominent Democratic lawmakers have targeted the FDA’s dealings with the drugmaker initiated well before the treatment was cleared in June. In a letter to the the Department of Health and Human Services on Tuesday, Rep. Katie Porter, D-California, charged Biogen with “undue influence” over the FDA’s review process.