The first new drug for Alzheimer’s disease in nearly 20 years was approved by state health officials on Monday. The decision ignores the warnings of independent advisers that the much-discussed treatment has not been shown to help slow brain-destroying disease.
The Food and Drug Administration has said it has approved a drug developed by Biogen for patients with Alzheimer’s disease. The medicine, known as Aduhelm, has the scientific name aducanumab (pronounced ‘add-yoo-CAN-yoo-mab’).
It is the only drug that US regulators say can cure the underlying disease instead of managing symptoms such as anxiety and insomnia. Shares of Biogen jumped after the announcement of the FDA, rising 38% to $ 395.85.
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. The FDA says it believes the benefits of the drug for people with Alzheimer̵
“We are aware of the attention associated with this approval,” said Dr. Patricia Cavazzoni, director of the FDA’s Center for Drug Evaluation and Research. “Ultimately, we decided to use the accelerated approval pathway – a pathway designed to provide earlier access to potentially valuable therapies for patients with serious illnesses when there is an unmet need.”
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.
$ 30,000 to $ 50,000 per year
Biogen did not immediately disclose the price, although analysts estimated that the drug could cost between $ 30,000 and $ 50,000 for a treatment worth a year.
A preliminary analysis by one group found that the drug would have to cost between $ 2,500 and $ 8,300 a year to be good value based on the “small overall health benefits” proposed by the company’s studies. The Institute for Non-Profit Clinical and Economic Review adds 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 necessary for memory, reasoning, communication and basic daily tasks. In the later stages of the disease, sufferers lose their 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.
The need for new drugs to treat the disease is “urgent,” Dr. Cavazzoni said. “Although the data for Aduhelm are complex in terms of its clinical benefits, the FDA has found that there is strong evidence that Aduhelm reduces amyloid beta plaques in the brain and that reducing these plaques is reasonably likely to predict important benefits for patients.”
Aducanumab 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 pipeline has been overwhelmed for years with failed Alzheimer’s treatment, representing billions in research costs. The FDA’s green light is likely to revive investment in similar therapies that have previously been postponed 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, 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 an external panel of FDA neurological experts. The group voted “no” to a series of questions about whether re-analyzed data from a study presented by Biogen showed that the drug was effective.
Cambridge, Massachusetts-Biogen stopped 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 the course, announcing 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 facts have made the results difficult to interpret, raising skepticism among many experts, including those at the FDA panel.