Executives debriefed analysts on the prospects for the medicine, the first drug to be approved in the US to slow Alzheimer’s disease, on a call last week following the release of the company’s third quarter 2021 financial results.
Aduhelm (aducanumab), to date, has not yet generated significant sales. In its first full quarter on the market, sales for the drug came to US$300K.
So far, the drug has only been adopted by 120 treatment centers in the US since its approval by the US Food and Drug Administration (FDA) in June. That number falls way short of the 900 sites the company had said it expected to be ready shortly after the green light by the regulators.
Biogen CEO, Michel Vounatsos, told investors on the call: “We are obviously disappointed with the delayed update of Aduhelm in the US.”
Reimbursement hurdles
But he stressed that the company, in this quarter, continued to progress the launch in the US in anticipation of the reimbursement decision for Medicare patients.
The core focus of Biogen in this context, said the chief executive, is ensuring increased patient access.
Alzheimer's affects 6.2 million Americans, a devastating illness that can have a profound impact on patients and their families (worldwide, around 35 million people are affected, according to the WHO).
The company is working through near-term challenges. “We have made steady progress on key metrics, but the healthcare system remains a major bottleneck,” said Vounatsos.
In particular, the lack of clarity on reimbursement has delayed patient access to the drug, said the chief executive.
Biogen is awaiting the upcoming national coverage determination (NCD), expected by next April, which, said the CEO, would clarify Medicare reimbursement for the entire class of antibodies directed against amyloids.
During the call, he that the developer is “working to improve the community’s understanding of our clinical data.”
Phase 3 clinical trial results for Aduhelm are currently under review at a “top-tier” journal, he added.
The company also intends to present data from EMBARK, a late-stage re-dosing study for patients who were previously enrolled in other Aduhelm trials, at an Alzheimer’s research conference early next month.
Review of approval procedures
The FDA's decision to approve Aduhelm has attracted criticism from many quarters.
The first therapy to target the fundamental pathophysiology of the disease, rather than the symptoms, data for the drug, as the FDA put it in June, are 'complicated'. The regulatory body backed the drug under the accelerated approval pathway, which requires a post-approval trial.
In August, the inspector general at the US Department of Health and Human Services (HHS) announced it was set to review how the FDA implements its accelerated approval pathway nearly two months after the approval of the Biogen drug.
Internal FDA memos, published in June, showed that the US regulatory body’s drug statistics office concluded that there was a lack of sufficient clinical trial data to put Biogen’s product on the market.
However, the released communications showed more senior officials decided that the evidence indicating that the drug worked, while inconclusive, supported accelerated approval (AA) of aducanumab for the treatment of patients with Alzheimer’s disease (AD).
Peter Stein, director of the FDA’s office of new drugs, in a memo detailing his support for the AA approach for Aduhelm, said he agreed with the agency’s Office of Neuroscience that the data was “not sufficiently compelling or persuasive to meet the substantial evidence standard for standard approval.”
Nonetheless, studies strongly suggested that treatment with aducanumab may result in clinical benefit, he argued.
