Biogen Inc. received approval for its controversial Alzheimer’s disease therapy, a landmark decision that stands to dramatically change treatment for the debilitating brain condition.
After decades of research failures by numerous companies, Biogen’s aducanumab became the first drug cleared by the U.S. Food and Drug Administration to slow the course of the mind-wasting disease that afflicts 6 million Americans.
The antibody therapy, which will be sold under the brand name Aduhelm, works by removing amyloid, a harmful protein that clogs the brains of Alzheimer’s patients. The last new drug treatment for the disease was first approved in 2003; existing therapies help with symptoms but don’t alleviate the underlying damage.
The FDA announced the decision in a statement on Monday.
“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,” the agency said.
The approval is one of the most consequential FDA decisions in recent years, and welcome news for millions of Americans with Alzheimer’s disease and their families. Patient advocacy groups have supported the drug’s clearance despite skepticism from some scientists about its mixed record in clinical trials.
The approval is also a significant victory for Cambridge, Massachusetts-based Biogen and its Tokyo-based partner Eisai Co. Biogen has seen wild swings in its share price with nearly every twist and turn of aducanumab’s development.
Now, it’s on the cusp of rolling out a product widely expected to be a blockbuster, even amid questions about the health-care system’s capacity to cope with demand.
Biogen shares were halted ahead of the news in New York trading and hadn’t yet resumed as of 11:26 a.m. in New York. U.S.-traded shares of Eisai leapt 42% to $105.21.
“This historic moment is the culmination of more than a decade of groundbreaking research in the complex field of Alzheimer’s disease,” said Biogen Chief Executive Officer Michel Vounatsos in an emailed statement.
Scientific Controversy
The long-awaited verdict is certain to be scrutinized. Scientists are still furiously debating amyloid’s role in the disease, and aducanumab’s clinical trial results were decidedly mixed.
In one giant trial conducted by Biogen, the drug was ineffective overall. A second, similar study found that high doses of the drug slowed progression of the disease by a modest 22% over 18 months.
The FDA approved the drug under its accelerated approval program, which means the companies will need to conduct further studies, and the agency could in theory remove it from the market if those studies don’t show a benefit.
For months, scientists and doctors have been bickering about which of the results to believe. Biogen and proponents of the medicine have argued that one trial succeeded because patients received higher doses than in the study that failed. Skeptics have said it’s just as likely that the successful trial was a fluke and argued that a third round is needed as a tiebreaker.
Annual sales could peak at $5 billion, analysts have said, providing a needed financial jolt to Biogen. Beyond the drug itself, health-care system expenses will include the eligibility tests, infusions and expensive scans to watch for side effects such as brain swelling. It’s unclear whether insurers will readily pay for the drug and all its associated costs.
Shares of Biogen competitors soared on the FDA approval.
Eli Lilly & Co. jumped as much as 13% to a record high. Lilly is working on its own medicine for the brain-wasting disease.