This image provided by Biogen on Monday, June 7, 2021 shows a vial and packaging for the drug Aduhelm. On Monday, June 7, 2021, the Food and Drug Administration approved Aduhelm, the first new medication for Alzheimer’s disease in nearly 20 years, disregarding warnings from independent advisers that the much-debated treatment hasn’t been shown to help slow the brain-destroying disease. (Biogen via AP)

U.S. approves much-debated Alzheimer’s drug aducanumab panned by experts

Drug from Biogen deemed “reasonably likely” to benefit Alzheimer’s patients

Government health officials in the United States on Monday approved the first new drug for Alzheimer’s disease in nearly 20 years, disregarding warnings from independent advisers that the much-debated treatment hasn’t been shown to help slow the brain-destroying disease.

The Food and Drug Administration said it approved the drug from Biogen based on results that seemed “reasonably likely” to benefit Alzheimer’s patients.

It’s the only therapy that U.S. regulators have said can likely treat the underlying disease, rather than manage symptoms like anxiety and insomnia.

The decision, which could impact millions of Americans and their families, is certain to spark disagreements among physicians, medical researchers and patient groups. It also has far-reaching implications for the standards used to evaluate experimental therapies, including those that show only incremental benefits.

The new drug, which Biogen developed with Japan’s Eisai Co., did not reverse mental decline, only slowing it in one study. The medication, aducanumab, will be marketed as Aduhelm and is to be given as an infusion every four weeks.

Dr. Caleb Alexander, an FDA adviser who recommended against the drug’s approval, said he was “surprised and disappointed” by the decision.

“The FDA gets the respect that it does because it has regulatory standards that are based on firm evidence. In this case, I think they gave the product a pass,” said Alexander, a medical researcher at Johns Hopkins University.

The FDA’s top drug regulator acknowledged that “residual uncertainties” surround the drug, but said Aduhelm’s ability to reduce harmful clumps of plaque in the brain is expected to help slow dementia.

“The data supports patients and caregivers having the choice to use this drug,” Dr. Patrizia Cavazzoni told reporters. She said FDA carefully weighed the needs of people living with the “devastating, debilitating and deadly disease.”

Under terms of the so-called accelerated approval, the FDA is requiring the drugmaker to conduct a follow-up study to confirm benefits for patients. If the study fails to show effectiveness, the FDA could pull the drug from the market, though the agency rarely does so.

Biogen said the drug would cost approximately $56,000 for a typical year’s worth of treatment, and said the price would not be raised for four years. Most patients won’t pay anywhere near that amount thanks to insurance coverage and other discounts. The company said it aims to complete its follow-up trial of the drug by 2030.

The non-profit Institute for Clinical and Economic Review said that “any price is too high” if the drug’s benefit isn’t confirmed in follow-up studies.

Some 6 million people in the U.S. and many more worldwide have Alzheimer’s, which gradually attacks areas of the brain needed for memory, reasoning, communication and basic daily tasks. In the final stages of the disease, those afflicted lose the ability to swallow. The global burden of the disease, the most common cause of dementia, is only expected to grow as millions more baby boomers progress further into their 60s and 70s.

Aducanumab (pronounced “add-yoo-CAN-yoo-mab”) helps clear a protein called beta-amyloid from the brain. Other experimental drugs have done that before but they made no difference in patients’ ability to think, care for themselves or live independently.

The pharmaceutical industry’s drug pipeline has been littered for years with failed Alzheimer’s treatments. The FDA’s greenlight Monday is likely to revive investments in therapies previously shelved by drugmakers.

The new medicine is manufactured from living cells and will be given via infusion at a doctor’s office or hospital.

Researchers don’t fully understand what causes Alzheimer’s but there’s broad agreement the brain plaque targeted by aducanumab is just one contributor. Evidence suggests family history, education and chronic conditions like diabetes and heart disease may all play a role.

“This is a sign of hope but not the final answer,” said Dr. Richard Hodes, director of the National Institute on Aging, which wasn’t involved in the Biogen studies but funds research into how Alzheimer’s forms. “Amyloid is important but not the only contributing factor.”

Patients taking aducanumab saw their thinking skills decline 22% more slowly than patients taking a placebo.

But that meant a difference of just 0.39 on an 18-point score of cognitive and functional ability. And it’s unclear how such metrics translate into practical benefits, like greater independence or ability to recall important details.

The FDA’s review of the drug has become a flashpoint in longstanding debates over standards used to evaluate therapies for hard-to-treat conditions. On one side, groups representing Alzheimer’s patients and their families say any new therapy — even one of small benefit — warrants approval. But many experts warn that greenlighting the drug could set a dangerous precedent, opening the door to treatments of questionable benefit.

The approval came despite a scathing assessment in November by the FDA’s outside panel of neurological experts. The group voted “no” to a series of questions on whether reanalyzed data from a single study submitted by Biogen showed the drug was effective.

Biogen halted two studies in 2019 after disappointing results suggested 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 showed the drug was effective at higher doses and the FDA had advised that it warranted review. Company scientists said the drug’s initial failure was due to some patients not receiving high enough doses to slow the disease.

But the changes to dosing and the company’s after-the-fact analysis made the results hard to interpret, raising skepticism among many experts, including those on the FDA panel.

The FDA isn’t required to follow the advice of its outside panelists and has previously disregarded their input when making similarly high-profile drug decisions.

About 600 U.S. medical specialists already prescribe the drug through Biogen’s studies and many more are expected to begin offering it. Many practical questions remain unanswered: How long do patients benefit? How do physicians determine when to discontinue the drug? Does the drug have any benefit in patients with more advanced dementia?

With FDA approval, aducanumab is certain to be covered by virtually all insurers, including Medicare, the government plan for seniors that covers more than 60 million people.

Even qualifying for the drug could be expensive. It’s only been tested in people with mild dementia from Alzheimer’s or a less severe condition called mild cognitive impairment. To verify a diagnosis could require brain scans that cost $5,000 or more. Insurers, including Medicare, don’t cover the scans because their benefits are unclear, but that could change if the scans become a gateway to treatment.

Additional scans will be needed to monitor potential side effects. The drug carries a warning about temporary brain swelling that can sometimes cause headaches, confusion and dizziness. Other side effects included allergic reactions, diarrhea and disorientation.

For patients in Biogen’s studies, the decision means they can continue taking a drug many believe has helped.

Kevin Bonham was diagnosed with early onset Alzheimer’s in 2016 after having difficulties with memory, reading and driving. The 63-year-old from Bear Creek Village, Pennsylvania, credits the drug with helping him keep his job as a mapmaker for another three years.

“Very quickly over a period of months it was like the fog was lifted from the top of my head,” said Bonham, who is now on disability and relies on his wife, Kim, to help care for him and his teenage daughter.

Like other trial participants, Bonham had to stop taking aducanumab in March 2019 after Biogen halted its trials. He resumed infusions nearly a year ago as part of the company’s ongoing research.

—Matthew Perrone, The Associated Press

RELATED: Alzheimer’s story from wife of Spirit of the West singer takes stage

Alzheimer's DiseaseHealthUSA

Just Posted

Police officers and their dogs undergo training at the RCMP Police Dog Services training centre in Innisfail, Alta., on Wednesday, July 15, 2015. Mounties say they are searching for an armed and dangerous man near a provincial park in northern Alberta who is believed to have shot and killed a service dog during a police chase. THE CANADIAN PRESS/Jeff McIntosh
RCMP search for armed man in northern Alberta after police dog shot and killed

Cpl. Deanna Fontaine says a police service dog named Jago was shot during the pursuit

Alberta now has 2,336 active cases of COVID-19, with 237 people in hospital, including 58 in intensive care. (Black Press file photo)
Red Deer down to 73 active cases of COVID-19, lowest since early November

The Central zone has 253 active cases of the virus

File photo
Update: Leduc RCMP request assistance to identify armed robbery suspect

Leduc RCMP are searching for suspect involved in an armed robbery at the Leduc Giant Tiger.

People line up to get their COVID-19 vaccine at a vaccination centre, Thursday, June 10, 2021 in Montreal. THE CANADIAN PRESS/Ryan Remiorz
Vaccines, low COVID case counts increase Father’s Day hope, but risk is still there

Expert says people will have to do their own risk calculus before popping in on Papa

Canadian Prime Minister Justin Trudeau is seen during a joint news conference following the EU-Canada Summit, in Brussels, Belgium, Tuesday June 15, 2021. Trudeau says Canada is on track now to have 68 million doses delivered by the end of July, which is more than enough to fully vaccinate all 33.2 million Canadians over the age of 12. THE CANADIAN PRESS/Adrian Wyld
Vaccine deliveries enough to fully vaccinate all eligible Canadians by end of July

Three in four eligible Canadians now have their first dose, nearly one in five fully vaccinated.

Chief Public Health Officer Theresa Tam listens to a question during a news conference, in Ottawa, Tuesday, Jan. 12, 2021. The number of confirmed COVID-19 cases attributed to the highly contagious Delta variant grew in Canada this week. THE CANADIAN PRESS/Adrian Wyld
Canada’s public health agency reports spike in confirmed cases of Delta variant

More than 2,000 cases of the variant confirmed across all 10 provinces and in one territory

The federal government says it wants to ban most flavoured vaping products in a bid to reduce their appeal to youth. THE CANADIAN PRESS/AP-Craig Mitchelldyer
Health Canada proposes ban on most vaping flavours it says appeal to youth

If implemented, the regulations would restrict all e-cigarette flavours except tobacco, mint and menthol

The Montreal Police logo is seen in Montreal on Wednesday, July 8, 2020. Some Quebec politicians are calling for an investigation after a video was released that appears to show a Montreal police officer with his leg on a young Black man’s neck during an arrest. THE CANADIAN PRESS/Paul Chiasson
Probe called for after video appearing to show Montreal officer’s knee on Black youth’s neck

Politicians call for investigation after clip evokes memories of George Floyd incident

Thousands of protesters make their way through the downtown core during a Black Lives Matter protest in Ottawa, Friday June 5, 2020. THE CANADIAN PRESS/Adrian Wyld
MPs’ study of systemic racism in policing concludes RCMP needs new model

Chair of the House public safety committee says it’s time for a reckoning on ‘quasi-military’ structure

A case filled with packages of boneless chicken breasts is shown in a grocery store Sunday, May 10, 2020, in southeast Denver. THE CANADIAN PRESS/AP-David Zalubowski
One million chickens euthanized during labour dispute at Quebec slaughterhouse

Premier says waste amounts to 13 per cent of the province’s chicken production thrown in the garbage

A section of the eastern slopes of the Canadian Rockies is seen west of Cochrane, Alta., Thursday, June 17, 2021. A joint federal-provincial review has denied an application for an open-pit coal mine in Alberta’s Rocky Mountains, saying its impacts on the environment and Indigenous rights aren’t worth the economic benefits it would bring. THE CANADIAN PRESS/Jeff McIntosh
Panel says Grassy Mountain coal mine in Alberta Rockies not in public interest

Public hearings on the project in southern Alberta’s Crowsnest Pass region were held last fall

Most Read