WASHINGTON – Government health officials on Monday approved the first new drug for Alzheimer’s disease in nearly 20 years, ignoring warnings from independent advisers that the highly controversial treatment failed to slow the destructive disease of the brain.
The Food and Drug Administration approved Biogen’s drug based on the results of a study showing that it appeared “reasonably likely” to benefit patients with Alzheimer’s disease. It’s the only therapy that U.S. regulators say can possibly treat the underlying disease, rather than managing symptoms like anxiety and insomnia.
The decision, which could impact millions of Americans and their families, is sure to spark disagreement among doctors, medical researchers and patient groups. It also has far-reaching implications for the standards used to evaluate experimental therapies, including those that only show incremental benefits.
The new drug, developed by Biogen with the Japanese company Eisai Co., did not reverse mental decline, only slowing it down in one study. The medicine, aducanumab, will be marketed as Aduhelm and should be given by infusion every four weeks.
Dr Caleb Alexander, an FDA adviser who recommended not to approve the drug, said he was “surprised and disappointed” by the decision.
“The FDA gets the respect it does because it has regulatory standards that are based on solid evidence. In this case, I think they gave the product a pass,” said Alexander, researcher. in medicine at Johns Hopkins University.
The FDA’s leading drug regulator has acknowledged that “residual uncertainties” surround the drug, but said Aduhelm’s ability to reduce harmful plaque buildup in the brain should help slow dementia.
“The data supports that patients and caregivers have a choice to use this drug,” Dr Patrizia Cavazzoni told reporters. She said the FDA carefully weighed the contribution of people living with the “devastating, debilitating and fatal disease.”
Under the so-called fast-track approval, the FDA is asking Biogen to conduct a follow-up study to confirm the benefits for patients. If the study does not show its effectiveness, the FDA could withdraw the drug from the market, although the agency rarely does.
Biogen said the drug would cost around $ 56,000 for a typical year of treatment, and said the price would not be increased for four years. Most patients won’t pay near that thanks to insurance coverage and other discounts. The company said it aims to complete the FDA-mandated follow-up trial by 2030.
Biogen shares jumped 38% in trading on Monday news, with analysts forecasting billions in future sales. The Cambridge, Massachusetts-based company plans to start shipping millions of doses within two weeks.
The nonprofit Clinical and Economic Review Institute, which studies the value of drugs, said Biogen’s drug would have to stop dementia completely to justify its price tag of $ 56,000 per year.
Some 6 million people in the United States and many more around the world suffer from 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 the ability to swallow. The global burden of disease, the most common cause of dementia, is only expected to increase as millions more baby boomers advance into their sixties and sixties.
Aducanumab (pronounced “add-yoo-CAN-yoo-mab”) helps remove a protein called beta-amyloid from the brain. Other experimental drugs did this, but they made no difference in the ability of patients to think, care for themselves, or live independently.
The pharmaceutical industry’s drug pipeline has been littered for years with failed treatments for Alzheimer’s disease. The FDA’s green light on Monday is likely to revive investment in therapies previously sidelined by drugmakers.
The new medicine is made from living cells and will be given by infusion in a doctor’s office or hospital.
Researchers don’t fully understand the causes of Alzheimer’s disease, but it is widely believed that the brain plaque targeted by aducanumab is only one contributor. Evidence suggests that family history, education, and chronic illnesses like diabetes and heart disease may all play a role.
“This is a sign of hope but not the definitive answer,” said Dr Richard Hodes, director of the National Institute on Aging, who was not involved in the Biogen studies but is funding research into how the disease of ‘Alzheimer’s is forming. “Amyloid is important but not the only contributing factor.”
Patients taking aducanumab saw their thinking skills decline 22% more slowly than patients taking placebo.
But that meant a difference of just 0.39 out of an 18-point cognitive and functional ability score. And it’s unclear how these measures translate into practical benefits, like greater independence or the ability to remember important details.
The drug’s review by the FDA has become a flashpoint in long-standing debates over the standards used to evaluate therapies for difficult-to-treat conditions. On the one hand, groups representing Alzheimer’s patients and their families argue that any new therapy, even of small benefit, deserves approval. But many experts warn that the drug’s green light could set a dangerous precedent, opening the door to treatments with questionable benefits.
The approval came despite a scathing assessment in November by the FDA’s External Neurological Expert Panel. The group voted “no” to a series of questions about whether reanalysed data from a single study submitted by Biogen showed the drug to be effective.
Biogen halted two studies in 2019 after disappointing results suggested that aducanumab would not meet its goal of slowing mental and functional decline in patients with Alzheimer’s disease.
A few months later, the company reverse course, announcing that a new analysis of a study showed that the drug was effective at higher doses and that the FDA had indicated that it deserved a review. Scientists at the company said the drug’s initial failure was due to some patients not receiving doses high enough to slow the disease.
But the dosage changes and the company’s afterthought analysis made the results difficult to interpret, sparking skepticism from many experts, including those on the FDA panel.
The FDA is under no obligation to follow the advice of its external panelists and has previously ignored their input when making equally important pharmaceutical decisions.
About 900 U.S. medical institutions are set to begin prescribing the drug, according to Biogen, with many more expected in the coming months. But key practical questions remain: how long do patients benefit from it? How do doctors decide when to stop the medicine? Does the drug have any benefit in patients with more advanced dementia?
With FDA approval, aducanumab is almost certain to be covered by most insurers, including Medicare, the government plan for the elderly that covers over 60 million people.
Insurers could try to manage the drug’s costs by requiring strict conditions, including brain scans to confirm the plaque, before agreeing to cover it.
Further testing will be needed to monitor for potential side effects. The medicine contains a warning about temporary swelling of the brain which can sometimes cause headache, confusion and dizziness. Other side effects included allergic reactions, diarrhea, and disorientation.
Although Biogen has studied the drug in people with mild dementia or early stage Alzheimer’s disease, the FDA label has approved the drug for anyone with Alzheimer’s disease, with a large population being since doctors have a great deal of latitude in diagnosing the disease.
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“The FDA empowers the doctor to make the diagnostic decision,” said Michel Vounatsos, CEO of Biogen, in an interview.
For patients already enrolled in Biogen trials, Monday’s announcement means they can continue to take a drug that many believe has helped.
Phillip Lynn, 63, was diagnosed with Alzheimer’s disease in the spring of 2017 after struggling with conversation and memory, including forgetting a recent vacation in Hawaii.
Her husband Kurt Rehwinkel says Lynn’s cognitive ability has stabilized since she started taking the Biogen drug over three years ago. And her performance on short-term memory tests actually improved, though the couple admit most patients are unlikely to see similar results.
“But even for those for whom it has little or no effect, I think hope is a good thing,” Rehwinkel said. “I don’t think false hopes exist.”