FDA Backpedals on Aduhelm Approval, Now Says Only Early Alzheimer’s Patients Should Get It

The Food and Drug Administration is altering its recent and controversial approval of a new drug for Alzheimer’s disease. The agency is now recommending that the drug, called Aduhelm and developed by Biogen, only be given to people in the earliest stages of the ultimately fatal neurodegenerative condition. The decision will greatly influence insurance coverage of the treatment, which is expected to cost $56,000 a year.

The move comes barely a month after the agency first approved the drug against the recommendations of an outside panel that reviewed the clinical trial evidence. Since then, researchers and lawmakers have criticized the FDA, with some calling the approval the worst decision of its kind made in recent history.

Among other things, critics have pointed to the lackluster data supporting Aduhelm’s benefits, with only one trial finding statistically significant evidence of slower cognitive decline in patients, and only in those who took the highest dose (the second trial didn’t find an overall slower decline but possibly some nonsignificant effect in high-dose patients).

Another major criticism of the FDA approval concerned the eligibility of the drug. Despite only being tested for people with mild cognitive impairment or early suspected Alzheimer’s, the FDA originally indicated it would be recommended for any patient, no matter how far along their disease was. The language fueled worries that broad use of the possibly ineffective drug would overwhelm public payers like Medicare, which provides health coverage for the majority of the estimated 5 million Americans currently living with Alzheimer’s. Now, the FDA is pulling back on its blanket endorsement.

“Since the agency approved Aduhelm, prescribers and other stakeholders have expressed confusion regarding the intended population for treatment,” FDA spokesperson Michael Felberbaum told the New York Times.

In its now updated label on prescribing the drug, the FDA recommends that Aduhelm be “initiated in patients with mild cognitive impairment or mild dementia stage of disease, the population in which treatment was initiated in clinical trials.” According to the NYT, the FDA sought the change, with Biogen agreeing to submit revised language. Previously, the company claimed that it would only market the drug to patients with early Alzheimer’s.

Doctors are allowed to prescribe drugs off-label, meaning that patients with more advanced Alzheimer’s disease may still gain access to Aduhelm. But the new labeling is almost certain to be widely adopted by insurance companies and payers like Medicare in determining coverage for Aduhelm.

This decision won’t settle the controversy over the drug’s approval, which some scientists fear will discourage research into other, potentially more effective treatments. More recently, STAT News has reported on a longstanding collaboration between certain FDA officials and Biogen before and during the approval process, which included discussions to submit the drug through a less rigorous pathway for approval. In response, lawmakers such as Rep. Katie Porter (D-CA) have called for a formal investigation into the FDA by the Office of the Inspector General, a government watchdog agency.