The US Food and Drug Administration (FDA) on Monday granted approval for the first new Alzheimer’s treatment since 2003.
The decision comes as the debate over whether to approve Biogen’s drug Aducanumab has stirred controversy, with some Alzheimer’s experts and an independent advisory committee opposing approval saying there’s not enough evidence that the drug is effective.
In a statement, a top FDA official on Monday said it decided to use an “accelerated approval” process to make drugs and therapies available to patients enduring “serious diseases where there is an unmet need” and “an expectation of clinical benefit.”
“In determining that the application met the requirements for Accelerated Approval, the Agency concluded that the benefits of Aduhelm for patients with Alzheimer’s disease outweighed the risks of the therapy,” Patrizia Cavazzoni, director of the FDA’s Center for Drug Evaluation and Research, said in the statement.
The agency called the demand for treatments “urgent” as Alzheimer’s affects more than 6 million Americans and is the sixth leading cause of death in the U.S.
Aducanumab, also known by the brand name Aduhelm, is considered the first treatment to register progress in removing protein deposits called amyloid beta plaques in the brain at the onset of Alzheimer’s, slowing cognitive decline.
The FDA usually follows its advisory committees’ recommendations, but this time it strayed from the Peripheral and Central Nervous System Drugs Advisory Committee’s recommendation in November to overwhelmingly reject Biogen’s drug.
With the accelerated approval, Biogen will be required to conduct another trial to prove the clinical benefit of its drug. The FDA could take steps to remove the drug from the market if the benefit is not proven.
“We believe that the data supports accelerated approval, while holding the company accountable for conducting an additional study to confirm the benefits observed in one of the trials, which we fully intend to do,” Cavazzoni told reporters on a press call Monday.
Two earlier clinical trials examined by the FDA produced conflicting results regarding the efficacy of the drug.
The FDA acknowledged it is “well-aware of the attention surrounding this approval,” noting the uncertainties in the data about the clinical benefit of the drug.
“There has been considerable public debate on whether Aduhelm should be approved,” the FDA said. “As is often the case when it comes to interpreting scientific data, the expert community has offered differing perspectives.”
Biogen’s chief executive Michel Vounatsos said in a statement that Aducanumab “will transform the treatment of people living with Alzheimer’s disease and spark continuous innovation in the years to come.”
The drugmaker, along with Japanese pharmaceutical company Eisai, announced a price for Aduhelm on Monday, saying the cost will amount to a yearly cost of about $56,000 per patient. The two partnering companies said they would not increase the price of Aduhelm over the next four years.
The FDA’s announcement on Monday drew mixed reactions from advocacy groups.
Alzheimer’s Association President and CEO Harry Johns celebrated the decision, calling it “a victory for people living with Alzheimer’s and their families.”
“It is a new day,” Johns said in a statement. “This approval allows people living with Alzheimer’s more time to live better. For families it means being able to hold on to their loved ones longer. It is about reinvigorating scientists and companies in the fight against this scourge of a disease. It is about hope.”
But Michael Carome, director of Public Citizen’s Health Research Group, condemned the FDA’s decision, arguing the “close collaboration” between Biogen and the agency “dangerously compromised” the agency’s review process.
He said the decision warrants an investigation by the Department of Health and Human Services Office of Inspector General.
“The FDA’s decision shows a stunning disregard for science and eviscerates the agency’s standards for approving new drugs,” Carome said in a statement. “Because of this reckless action, the agency’s credibility has been irreparably damaged.”
The Hill has reached out to Biogen and the FDA for comment.
Rep. Cathy McMorris Rodgers (R-Wash.), the ranking member of the House Energy & Commerce Committee, tweeted that the approval gives patients and their families “renewed hope.”
She argued that the Democrats’ bill to allow Medicare to negotiate prescription drug prices, H.R. 3, would inhibit innovation and prevent new medicines similar to Aduhelm from reaching the public.
“We need more biomedical innovation so patients have a fighting chance,” she posted. “That’s why we must stop Speaker Pelosi’s radical drug pricing scheme for #FewerCures. It would halt innovation in its tracks.
Democrats, including Speaker Nancy Pelosi (D-Calif.), assert that negotiated prices would make drugs more accessible and affordable.