sunny.png
Saturday June 12th, 2021 6:52PM

FDA approves much-debated Alzheimer’s drug panned by experts

By The Associated Press
Related Articles
  Contact Editor

WASHINGTON (AP) — Government health officials 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 approved the drug from Biogen based on study results showing it 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 the FDA carefully weighed the input of people living with the “devastating, debilitating and deadly disease.”

Under terms of the so-called accelerated approval, the FDA is requiring Biogen 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 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 Monday on the news, with analysts forecasting billions in future sales. The Cambridge, Massachusetts-based company plans to begin shipping millions of doses within two weeks.

The non-profit Institute for Clinical and Economic Review, which studies drug value, said Biogen's drug would have to halt dementia entirely to justify its $56,000 per-year price tag.

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 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 study 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 900 U.S. medical facilities are ready to begin prescribing the drug, according to Biogen, with many more expected in coming months. But key practical questions remain: 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 almost certain to be covered by most insurers, including Medicare, the government plan for seniors that covers more than 60 million people.

Insurers could try to manage the drug’s costs by requiring strict conditions, including brain scans to confirm plaque, before agreeing to cover it.

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.

Although Biogen studied the drug in people with mild dementia or early-stage Alzheimer's, the FDA label approved the drug for anyone with Alzheimer's, a sweeping population given doctors have broad leeway in diagnosing the condition.

“The FDA is empowering the physician to make the decision on diagnosis,” Biogen CEO Michel Vounatsos said in an interview.

For patients already enrolled in Biogen's trials, Monday's announcement means they can continue taking a drug many believe has helped.

Phillip Lynn, 63, was diagnosed with Alzheimer’s in the spring of 2017 after having trouble with conversation and memory, including forgetting a recent vacation to Hawaii.

His husband Kurt Rehwinkel says Lynn's cognitive ability has stabilized since starting on Biogen’s drug more than three years ago. And his performance on short-term memory tests has actually improved, though the couple acknowledges most patients are unlikely to see similar results.

“But even for those who it has little or no effect, I think hope is a good thing,” said Rehwinkel. “I don’t think there’s such a thing as false hope.”

___

Follow Matthew Perrone on Twitter: @AP_FDAwriter

___

AP writers Lauran Neergaard and Tom Murphy contributed to this report.

___

The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute’s Department of Science Education. The AP is solely responsible for all content.

  • Associated Categories: U.S. News, Associated Press (AP), AP National News, AP Online National News, Top U.S. News short headlines, Top General short headlines, AP Health, AP Business, AP Business - Corporate News, AP Health - Senior Health, AP Business - Industries, AP Business - Health Care
© Copyright 2021 AccessWDUN.com
All rights reserved. This material may not be published, broadcast, rewritten, or redistributed without permission.
FDA approves much-debated Alzheimer’s drug panned by experts
Government health officials have approved the first drug that they say may help slow Alzheimer’s disease
11:16AM ( 4 minutes ago )
Supreme Court agrees to hear 2nd 'state secrets' case
The Supreme Court has agreed to decide whether a lawsuit can go forward in which a group of Muslim residents of California allege the FBI targeted them for surveillance because of their religion
11:12AM ( 7 minutes ago )
Jeff Bezos riding his own rocket in July, joining 1st crew
Jeff Bezos will ride his own rocket into space next month, joining the first crew to fly in a Blue Origin capsule
11:04AM ( 15 minutes ago )
Associated Press (AP)
Harris to address migration as she meets Guatemalan leader
Vice President Kamala Harris faces a major test of her diplomatic skills when she meets with Guatemala's president as part of her efforts to address the root causes of migration to the U.S. from the region
9:59AM ( 1 hour ago )
High court won't review men-only draft registration law
The Supreme Court says it won’t take up a case that asked it to decide whether it’s sex discrimination for the government to require only men to register for the draft when they turn 18
9:54AM ( 1 hour ago )
German state vote triumph boosts Merkel's center-right bloc
Chancellor Angela Merkel’s center-right bloc is claiming momentum for the election in September that will choose Germany’s next leader after a larger-than-expected win in the last regional ballot before the nation votes
9:41AM ( 1 hour ago )
AP National News
High court asked to review men-only draft registration law
The Supreme Court is being asked to decide whether the government can continue to require men alone, and not women, to register for the draft when they turn 18
12:46AM ( 10 hours ago )
Oldest male chimpanzee in US dies at San Francisco zoo
The oldest male chimpanzee living in an accredited North American zoo died Saturday at the San Francisco Zoo & Gardens
11:55PM ( 11 hours ago )
Cleared Chicago priest holds first Mass since reinstatement
An activist Roman Catholic priest cleared by an Archdiocese of Chicago investigation into decades-old sexual abuse claims has returned to lead worship at his longtime Chicago church
10:48PM ( 12 hours ago )
Top U.S. News short headlines
India to provide free vaccines to all in major policy shift
Indian Prime Minister Narendra Modi says his government will provide free coronavirus shots to any adult starting later this month and will take back control of the country’s vaccination drive, marred by delays and shortages
10:02AM ( 1 hour ago )
The Latest: Moderna seeks to use vaccine for adolescents
Moderna Inc. has filed a request with the European Medicines Agency for its COVID-19 vaccine to be granted authorization for use in adolescents
9:14AM ( 2 hours ago )
The Latest: Slovakia becomes 2nd EU nation to use Sputnik V
Slovakia has become the second European Union country to administer the Russian-made Sputnik V vaccine
5:50AM ( 5 hours ago )
AP Health
Stocks wobble in early trading, pull back from record highs
Stocks were listless in early trading Monday after briefly approaching the record highs they reached a month ago
10:26AM ( 54 minutes ago )
US stocks edge mostly higher, hovering near record highs
Stocks are nudging mostly higher in early trading, putting the S&P 500 and the Dow Jones Industrial Average back near the record highs they reached a month ago
9:43AM ( 1 hour ago )
AP Business
‘Conjuring 3’ tops ‘A Quiet Place 2’ as moviegoing returns
After its triumphant first weekend, “A Quiet Place Part II” fell 59% at the North American box office leaving room for the third movie in the “Conjuring” franchise to take first place
1:31PM ( 21 hours ago )
G-7 back steps to deter tax dodging by multinational firms
The Group of Seven wealthy democracies have agreed to support a global minimum corporate tax of at least 15% to deter multinational companies from avoiding taxes by stashing profits in low-rate countries
2:47PM ( 1 day ago )
G-7 nations sign key pact to make tech giants pay fair taxes
The Group of Seven wealthy democracies have agreed to support a global minimum corporate tax rate of at least 15% in order to deter multinational companies from avoiding taxes by stashing profits in low-rate countries
10:00AM ( 2 days ago )
AP Business - Corporate News
NFL pledges to halt 'race-norming,' review Black claims
The NFL is pledging to halt the use of “race-norming” in the $1 billion settlement of brain injury claims and to review Black claims for any potential race bias
3:37PM ( 4 days ago )
Despite vaccines, nursing homes struggle with outbreaks
Nursing homes across the country are still reporting scattered COVID-19 outbreaks and COVID-associated deaths, albeit at much smaller rates than during the height of the pandemic
1:53PM ( 5 days ago )
The Latest: France offers vaccine jabs to all adults
France has opened up virus vaccinations to adults of all ages, earlier than originally scheduled, as vaccine deliveries have picked up speed
11:59AM ( 6 days ago )
AP Health - Senior Health
Pipeline foes gear up for large northern Minnesota protests
Environmental and tribal groups opposed to Enbridge Energy’s ongoing effort to replace its aging Line 3 crude oil pipeline are planning large protests in northern Minnesota as the Canadian-based company gears up for a final construction push
4:39PM ( 18 hours ago )
Pipeline foes gear up for large northern Minnesota protest
Environmental and tribal groups opposed to Enbridge Energy’s ongoing effort to replace its aging Line 3 crude oil pipeline are planning large protests in northern Minnesota as the Canadian-based company gears up for a final construction push
4:11PM ( 19 hours ago )
The Latest: Sri Lanka receives 1M doses of Sinopharm vaccine
Sri Lanka has received one million doses of China’s Sinopharm vaccines amid the island nation’s recent surge in infections and COVID-19 vaccine shortage
11:33AM ( 23 hours ago )
AP Business - Industries
The Latest: Turkey’s single-day coronavirus deaths below 100
Turkey’s single-day COVID-19 death toll has dropped below 100 for the first time in more than two months
2:00PM ( 2 days ago )
The Latest: Taiwan receives 1.2M vaccine doses from Japan
A flight carrying 1.24 million doses of AstraZeneca coronavirus vaccine from Japan has touched down in Taiwan
10:56AM ( 3 days ago )
The Latest: Hungary to end govt-run mass vaccination drive
The Hungarian government says it is suspending the country’s mass vaccination drive and reducing the number of places coronavirus shots will be available
8:55AM ( 3 days ago )
AP Business - Health Care
Supreme Court agrees to hear 2nd 'state secrets' case
The Supreme Court has agreed to decide whether a lawsuit can go forward in which a group of Muslim residents of California allege the FBI targeted them for surveillance because of their religion
11:12AM ( 7 minutes ago )
Jeff Bezos riding his own rocket in July, joining 1st crew
Jeff Bezos will ride his own rocket into space next month, joining the first crew to fly in a Blue Origin capsule
11:04AM ( 16 minutes ago )
To the beach! Spain opens borders to tourists, cruise ships
Spain is jumpstarting its summer tourism season by welcoming vaccinated visitors from most countries as well as European visitors who can prove they are not infected with coronavirus
10:52AM ( 27 minutes ago )
Train barrels into another in Pakistan, killing at least 45
Authorities say an express train barreled into another that had derailed in Pakistan before dawn, killing at least 45 people
10:50AM ( 30 minutes ago )
Greece says many migrants in Turkey could seek asylum there
Greece has designated neighboring Turkey as a safe country in which to seek asylum for the vast majority of asylum-seekers departing its shores for Greece
10:43AM ( 36 minutes ago )