(AP) — The government approved the first new Alzheimer's drug in nearly 20 years on Monday, despite warnings from independent advisers that the much-debated treatment hasn't been shown to help slow the brain-destroying disease.
and Drug Administration announced that it had approved Biogen
's drug for Alzheimer's disease patients.
It is the only drug that U.S. regulators have said is likely to treat the underlying disease rather than manage symptoms such as anxiety and insomnia.
The decision, which could affect millions of older Americans and their families, is sure to spark debate among physicians, medical researchers, and patient advocacy groups, and it has far-reaching implications for the standards used to evaluate experimental therapies, including those that show only incremental benefits.
In one study, the new drug, developed by Biogen in collaboration with Japan
's Eisai Co., did not reverse mental decline but only slowed it. It is administered as an infusion every four weeks.
The FDA is requiring the drugmaker to conduct a follow-up study to confirm the drug's benefits for patients; if the study fails to show efficacy, the FDA has the authority to remove the drug from the market, though this is rare.
Biogen did not immediately disclose the price, but analysts estimate the drug will cost between $30,000 and $50,000 for a year's supply.
Based on the “small overall health
gains” suggested by company studies, one group determined that the drug would need to be priced between $2,500 and $8,300 per year to be a good value. The non-profit Institute for Clinical and Economic Review added that “any price is too high” if the drug’s benefit is not confirmed in follow-up studies.
Alzheimer's disease affects nearly 6 million people
in the United States
and many more around the world, gradually attacking areas of the brain required for memory, reasoning, communication, and basic daily tasks. In the final stages of the disease, those affected lose the ability to swallow. The global burden of the disease, the most common cause of dementia, is expected to grow as millions more Baby Boomers progress.
Aducanumab (pronounced “add-yoo-CAN-yoo-mab”) is an experimental drug that aims to help clear harmful clumps of a protein called beta-amyloid from the brain. Other experimental drugs have done so in the past, but they made no difference in patients’ ability to think, care for themselves, or live independently.
For years, the pharmaceutical industry
's drug pipeline
has been littered with failed Alzheimer's treatments, costing billions of dollars in research, and the FDA's approval is expected to resurrect investments in similar therapies that had previously been shelved by drugmakers.
The new medicine is made from living cells and must be administered via infusion at a doctor's office or hospital. The most common side effects were inflammation in the brain, but most cases did not result in symptoms or long-term problems.
The FDA’s review of the drug has become a flashpoint in long-running debates over the standards used to evaluate therapies for difficult-to-treat conditions. On one side, groups representing Alzheimer’s patients and their families argue that any new therapy — even one with minor benefits — warrants approval. However, many experts have warned that approving the drug could set a dangerous precedent, opening the door to other drugs with similar benefits.
The approval came despite a harsh assessment in November by the FDA's outside panel of neurological experts, who voted "no" to a series of questions about whether reanalyzed data from a single study submitted by Biogen demonstrated that the drug was effective.
Cambridge, Massachusetts-based Biogen halted two studies of the drug in 2019 after disappointing results suggested that aducanumab would not meet its goal of slowing mental and functional decline in Alzheimer's patients. Several months later, the company reversed course, announcing that a new analysis of one of the studies revealed that the drug was effective at higher doses and that the FDA had advised that the drug be approved.
However, dosing changes and the company's post-mortem analysis made the results difficult to interpret, raising the skepticism of many experts, including those on the FDA panel.
The Howard Hughes Medical Institute's Department of Science Education
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