As FDA Narrows Use of New Alzheimer’s Drug, Providers Proceed With Caution

Since the accelerated approval of an Alzheimer's drug called Aduhelm, Santosh Kesari, MD, PhD, has been getting calls from patients of all stages of the disease.

“Right now, we're having patients from ‘no Alzheimer’s’ to 'mild Alzheimer's’ to ‘late Alzheimer's’ calling for the drug,” Kesari, who is the director of neuro-oncology at Providence Saint John’s Health Center and chair of the Department of Translational Neurosciences and Neurotherapeutics at Saint John’s Cancer Institute, tells Verywell.

But the drug is no longer intended for all stages for Alzheimer's. On Thursday, the Food and Drug Administration (FDA) announced a change in the labeling of Aduhelm (aducanumab), specifying that the drug is for patients with mild cognitive impairment or mild dementia stage of disease. It is not for people with later stages of Alzheimer’s, since they were not included in clinical trials.

Previous labeling did not specify that the drug was only for patients in early stages of the disease, and the FDA initially told reporters that the drug was “relevant to all stages of Alzheimer’s disease,” according to the Associated Press.

“That was a mistake on their part, because the drug was studied in early cases,” Kesari says. “For patients who are really bad off—in nursing homes; requiring a lot of assistance—it hasn't been studied there, and it shouldn’t be used there.”

Aduhelm’s manufacturer, Biogen, initially announced the accelerated approval in a press release on June 7. It is the first new drug for Alzheimer’s in almost 20 years.

What makes Aduhelm unique is its potential to stop the trajectory of the disease, rather than just manage symptoms. In a clinical trial with three, double-blind studies, the drug reduced the appearance of beta-amyloid plaque—a protein that builds up in people with Alzheimer’s disease—among patients who received it, according to the FDA.

Still, experts have doubts about the drug’s effectiveness.

Andrew Peterson, MA, PhD, says that he has doubts about the drug's effectiveness, and fears that the change in labeling could complicate things for physicians. 

"Clinicians will now have to have difficult conversations about risks and may feel ethically torn about whether they should prescribe,” Peterson, assistant professor in the Department of Philosophy and Institute for Philosophy and Public Policy at George Mason University, writes to Verywell. “The change in the prescription instructions is going to make these conversations harder—not easier—especially for off-label prescriptions."

Further, to what extent Medicare and private insurance companies will cover the cost of Adulhelm treatments, which come at the steep price of $56,000 a year, is unclear. The FDA has not guaranteed that Medicare will cover treatment costs.

"It carries significant risks and it has a hefty price tag,” Peterson adds. “This is further evidence that something has gone seriously wrong with the FDA approval process."

Kesari also works in oncology, where he says accelerated drug approvals are common.

“In oncology, we're used to accelerated approval because those are bad diseases where patients decline very quickly,” he says. “But in neurology, this is a kind of a unique situation. [Alzheimer’s is] not a fast-moving disease, so it's really a risk/benefit analysis."

For now, he says it is important for providers to treat the drug with caution and for specific use, prioritizing one-on-one patient evaluations. 

“There's still a lot of unanswered questions,” he says. “We're preparing to make it available, but deliberating on what it really is: an accelerated approval, not a full approval. We shouldn't be rushing to treat everyone and we should be careful about which patients [to treat, and] what their risk factors are.”