A make-or-break moment is quickly approaching for what some consider the world's most closely watched experimental medicine. By June 7, the Food and Drug Administration should decide whether to approve or reject aducanumab, a potentially first-of-its-kind therapy meant to slow the progression of Alzheimer's disease.
The FDA's verdict is far from certain. The data supporting aducanumab are complex and controversial. Alzheimer's experts remain torn on whether the drug actually improves patients' lives. Even the FDA's internal staff are divided about the prospect of approval.
However the agency ultimately leans, its decision will have significant impacts on both Alzheimer's care as well as aducanumab's developers, Biogen and Eisai.
As they wait to hear from the FDA, executives at Biogen, which is responsible for marketing aducanumab in the U.S, are preparing for what would be the most important drug launch in the company's history. In February, they said Biogen plans to spend about $600 million on the launch, with the initial aim of targeting several hundred of the "most important" Alzheimer's treatment centers that serve a high volume of patients.
And on Thursday, during a conference call on the company's first quarter earnings, executives shed more light on how they intend to address some of the challenges that would come with selling a drug like aducanumab.
Alzheimer's doctors are bracing for aducanumab, if it comes to market, to be met with high demand from patients and caregivers, as there are currently no available treatments to reduce the cognitive decline caused by the disease. With several million Alzheimer's patients in the U.S., such demand could overwhelm treatment centers.
That's one problem Biogen's been trying to get ahead of, according to CEO Michel Vounatsos.
"We know that the availability of specialists and diagnosis capabilities are a bottleneck, so we had to prepare the sites of care," he said on a Thursday call with investors. "Today, we anticipate approximately 600 [are] ready to treat, but many more are [in] the works."
"We are pleased with where we are, in terms of those sites and their ability to welcome the patients, to diagnose the patients, to dose the patients, to monitor the patients," he added.
But doctors also expect many patients won't be able to access aducanumab — either because of restrictions put in place by the FDA, or because of pushback from insurance companies over what's expected to be a high-priced therapy.
If the FDA, for example, requires patients to undergo brain scans to monitor for the potential side effects associated with aducanumab, that could be a obstacle in the drug's launch.
"This drug, to be delivered safely, you have to do imaging repeatedly. That's just hard to do," Mary Sano, director of the Alzheimer's Disease Research at Mount Sinai School of Medicine, told BioPharma Dive early this year.
"I think the safety issues, the monitoring issues, the diagnostic issues, might relegate these drugs to become boutique drugs for the super-specialists like myself," Marwan Sabbagh, director of the Lou Ruvo Center for Brain Health at Cleveland Clinic Nevada, said in a January interview. "I know that limits access, because there's so few of us super-specialists."
Al Sandrock, Biogen's head of research and development, noted on Thursday's call that in the late-stage clinical testing of aducanumab, patients underwent six to nine MRI scans. But he said he doesn't think as many would be needed outside of clinical testing.
Exactly how many scans are required is to be determined, according to Sandrock, as that figure depends on labeling discussions with the FDA. Still, "virtually every center, even private practice clinics, have ready access to MRI. And as I said it's a standard sequence. I don't think it's going to be a major problem to monitor for [side effects]."
On price, Vounatsos said in February that his team was still working on aducanumab's potential price tag. But it appears that they've made progress.
"I think that we are there," he said Thursday, referring to price. "We have done a thorough engagement with different stakeholders, considering the burden of the disease and the clinical meaningfulness that aducanumab will bring."
Vounatsos added that Biogen has engaged with organizations which evaluate drug pricing, including the Institute for Clinical and Economic Review, to better inform its decision.
Should aducanumab gain approval, Biogen will be under immense pressure to pull off a successful launch. The company has been banking on the drug to offset growing problems elsewhere in its business. From January to March, Biogen's total revenue was down 25% compared to the same period a year prior.