In a meeting Friday, outside advisers to the Food and Drug Administration took a dim view of studies supporting what could be the first new drug for Alzheimer’s disease in nearly 20 years.
Over more than six hours, an 11-member advisory committee of outside experts raised numerous concerns about the data underpinning aducanumab, a drug developed in a partnership between Cambridge, Massachusetts-based Biogen and Japan-based Eisai.
In a final vote, 10 members of the advisory committee panel said the studies so far did not support the drug’s effectiveness, though FDA had argued otherwise in a presentation the panel. The 11th member registered an “uncertain” vote. Though the vote is nonbinding, as is customary, FDA will consider the panel’s recommendations as the agency reviews the drug’s application for approval.
In a statement sent to MedCity News, Biogen’s CEO thanked patients and others for speaking at the FDA meeting.
“Biogen thanks the many patients and advocates who shared their personal thoughts and experience at today’s Advisory Committee meeting, reflecting the significant unmet need for a treatment for Alzheimer’s,” Michel Vounatsos said. “We appreciated the opportunity to share our data with the Advisory Committee, and we will continue to work with the FDA as it completes its review of our application.”
Aducanumab aims to slow the build-up of amyloid beta plaque in the brain, a key feature of Alzheimer’s disease. Previous therapies with the same goal have largely proven futile.
The drug from Biogen and Eisai appeared headed for the same dustbin in spring 2019. The companies halted a pair of clinical trials after it was determined they were likely to fail. The trials involved patients with mild cognitive impairment and dementia due to Alzheimer’s.
But the drug gained new life in October 2019 when Biogen unveiled data from the trials showing the drug appeared to work when taken in higher doses.
Patients and caregivers acknowledged the drug may not be perfect. But they said it represented hope for people suffering from Alzheimer’s and a spur for continued research in the field.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
One speaker – Dr. Stephen Salloway, an Alzheimer’s researcher and professor at the Warren Alpert Medical School of Brown University in Rhode Island – compared aducanumab to AZT, an early treatment for HIV/AIDS that eventually gave way to more effective drugs.
“We are at a critical juncture in Alzheimer’s disease,” Salloway told the panel, adding that patients and their families don’t have time to wait another four or five years for more clinical studies.
Other speakers said they had taken part in clinical trials for aducanumab and that it had helped them.
FDA staff also took a positive view of the drug. In materials prepared ahead of the meeting Friday, the agency described the study involving higher doses as being “highly persuasive and capable of providing the primary contribution to a demonstration of substantial evidence of effectiveness of aducanumab.”
Scientists on the FDA advisory panel were largely unconvinced. They recognized the urgent need for new treatments for the fatal disease, which afflicts 5.8 million Americans and has no cure. But they were concerned about the precedent if they approved a drug based on studies they deemed inconclusive at best.
“I think there’s a huge danger to approving something that turns out not to be effective. I think that danger is much, much greater,” said panel member Dr. Joel S. Perlmutter, a neurology professor at the Washington University School of Medicine in St. Louis.
Photo: wildpixel, Getty Images
Update: This story has been updated with comment from Biogen
