The Food and Drug Administration approved the drug for Alzheimer’s disease on Monday, the agency approving a new therapy for the disease for the first time since 2003. The move comes after an independent advisory committee in November called on the agency to reject a drug called vivisanumab, warning that treatment has not been shown to help slow the progression of the disease.
Aducanumab is the only drug that US regulators say can treat the underlying disease instead of managing symptoms such as anxiety and insomnia. It is manufactured by Massachusetts-based company Biogen and will be sold under the name Aduhelm.
The decision, which could affect millions of elderly Americans and their families, is sure to spark disagreements between doctors, medical researchers and patient groups. It also has far-reaching implications for the standards used to evaluate experimental therapies, including those that show only additional benefits.
Maria Carillo, chief scientific director of the Alzheimer̵
“The Alzheimer’s Association believes that approval is ushering in a new era in Alzheimer’s treatment and even in research,” she said. “For people living with Alzheimer’s disease, this treatment means the potential to slow the decline.”
The FDA acknowledged the controversy over the drug in a statement by Dr. Patrizia Cavazzoni, director of the FDA’s Center for Drug Evaluation and Research.
“There has been considerable public debate over whether Aduhelm should be approved. As is often the case when it comes to interpreting scientific data, the expert community offers different points of view,” she wrote, adding that “the data included in the candidate’s opinion , were highly complex and left a residual uncertainty as to the clinical benefit. “
During a media briefing Monday, Cavazzoni explained that the drug was approved by the FDA’s Accelerated Path, an approach that allows the FDA to approve drugs for serious or life-threatening illnesses when the drug “has shown improvement in the surrogate endpoint which is considered reasonably likely to predict clinical benefit. “
In the case of Aduhelm, the surrogate endpoint is the reduction of amyloid plaque in the brain, lumps of protein that are considered a hallmark of the disease. Reducing these plaques “is reasonably likely to predict the clinical benefit of the drug,” she said.
However, the fact that a drug is likely to predict clinical benefit does not mean that it is guaranteed. Therefore, FDA-approved drugs must continue to be tested in the drug to confirm its clinical benefits.
“If the clinical benefit is not confirmed or if the study is not conducted in a timely manner, we can withdraw the approval of this therapy,” Cavazzoni said.
The benefits need to be confirmed
Dr. Jason Karlavish, co-director of the Penn Memory Center in Philadelphia and a field researcher for clinical trials of the Biogen drug, disagreed with the FDA’s decision.
“This will be a real challenge for patients and caregivers and clinicians whether to prescribe the drug,” he said. “It will also challenge researchers who are experimenting and planning attempts to develop better drugs.”
Still, Karlavish said he would prescribe Vismanumab, but only “after talking to the patient and his family about the noticeable uncertainties surrounding whether the drug even works.”
The new drug, which Biogen developed with Japan’s Eisai Co., did not reverse the mental decline, but only slowed it down in one study. The drug is given as an infusion every four weeks.
The FDA requires the drug manufacturer to conduct a follow-up study to confirm the benefits of the drug for patients. If the study fails to show effectiveness, the FDA may withdraw the drug from the market, although the agency rarely does.
Biogen did not immediately disclose the price, although analysts estimated that the drug could cost $ 30,000 to $ 50,000 for a treatment worth a year.
Preliminary analysis by one group found that the drug would have to be priced at $ 2,500 to $ 8,300 a year to be good value based on the “small overall health benefits” proposed by the company’s studies. The non-profit Institute for Clinical and Economic Review added that “any cost is too high” if the benefits of the drug are not confirmed in subsequent studies.
Nearly 6 million people in the United States and many more around the world have Alzheimer’s disease, which gradually attacks areas of the brain needed for memory, reasoning, communication and basic daily tasks. In the later stages of the disease, sufferers lose the ability to swallow. The global severity of the disease, the most common cause of dementia, is expected to increase with the advancement of millions more Baby Boomers in their 60s and 70s.
Aducanumab (pronounced “add-yoo-CAN-yoo-mab”) aims to help clear harmful lumps of a protein called beta-amyloid from the brain. Other experimental drugs have done this before, but have made no difference in patients’ ability to think, take care of themselves, or live independently.
The pharmaceutical industry’s drug pipeline has been overwhelmed for years with failed Alzheimer’s treatment, which represents billions in research costs. The FDA’s green light is likely to revive investment in such therapies previously delayed by drug manufacturers.
The new drug is made from living cells that will need to be administered by infusion in a doctor’s office or hospital. The most common side effects are inflammation in the brain, but most cases do not cause symptoms or permanent problems.
The FDA’s review of the drug has become a flame in the long-running debate over the standards used to evaluate therapies for difficult-to-treat conditions. On the one hand, groups representing Alzheimer’s patients and their families say that any new therapy – even a small benefit – requires approval. But many experts warn that green lighting the drug could set a dangerous precedent by opening the door to treatments of dubious benefit.
The approval came despite a sharp assessment in November by the FDA’s outer panel of neurological experts. The group voted “no” to a series of questions on whether re-analyzed data from a single study presented by Biogen showed that the drug was effective.
Cambridge, Massachusetts-based Biogen halted two trials of the drug in 2019 after disappointing results suggested that visvanumab would not meet its goal of slowing mental and functional decline in Alzheimer’s patients. A few months later, the company reversed course. that a new analysis of one of the studies showed that the drug was effective at higher doses and that the FDA advised that a review was justified. The company’s researchers said that the initial refusal of the drug was due to the fact that some patients did not receive high enough doses to slow down the disease.
But changes to the company’s dosage and analysis of the facts have made the results difficult to interpret, raising skepticism among many experts, including those at the FDA panel.
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