|
 In a field littered with unrelenting failure, Biogen believes in
aducanumab it has the first drug that can treat an underlying cause,
and therefore slow progression, of Alzheimer's. But its path to
approval has been anything but smooth or assured.
Biogen abruptly ended clinical trials of aducanumab last year after
an early look at trial results showed it was not effective. Last
October, the company shocked many Alzheimer’s experts by reversing
course, saying that a new analysis showed aducanumab could help
patients with early-stage disease preserve their ability to function
independently for longer. In July, Biogen filed for approval from
the Food and Drug Administration.
Now the agency faces tremendous pressure to approve a treatment
option for millions of Americans suffering from Alzheimer's and the
millions more expected to face it in coming years.
Patient advocates say the need for a new Alzheimer’s treatment that
could help people remain independent is heightened by the
coronavirus pandemic, which has killed more than 229,000 people in
the United States, including tens of thousands of seniors in nursing
homes.
"The pandemic came and it changed everything,” said Russ Paulsen,
chief operating officer at patient advocacy group
UsAgainstAlzheimer's. “We need something to keep people out of
nursing homes."
A committee of outside advisers to the FDA will discuss aducanumab
on Nov. 6. The agency's final decision is expected by March.
European health regulators have also accepted the drug for review.
Charles Flagg, a 79-year-old retired minister from Jamestown, Rhode
Island, had been enrolled for years in a trial of aducanumab before
it was stopped. He started receiving the drug again in August as
part of a follow-up study, according to his wife Cynthia Flagg.
“He's not 100 percent himself, but overall I'm not dealing with
someone that needs to be led around or be in a care home,” Flagg
said.
Aducanumab, an antibody designed to remove amyloid plaques from the
brain - a strategy tried with many failed Alzheimer's drugs - would
reap billions of dollars in sales if approved.
Biogen, along with partner Eisai Co Ltd <4523.T>, is one of the last
large drugmakers pursuing treatments for a disease that afflicts
nearly 6 million Americans and millions more worldwide. Biogen
estimates about 1.5 million people with early Alzheimer’s in the
United States could be candidates for the drug.
[to top of second column] |
'CLINICALLY MEANINGFUL'
Late last year, Biogen said one of its two pivotal studies of aducanumab showed
a statistically significant benefit at slowing cognitive and functional decline
in patients with early Alzheimer's. A second trial failed to achieve that goal,
but did show a benefit for a subset of patients who were given a high dose for
at least 10 months.
In March, it opened a follow-up long-term safety study to 2,400 people who had
previously participated in trials of aducanumab, like Flagg.
Many Alzheimer's researchers say Biogen should conduct a third large study to
prove aducanumab works. They worry about its possible side-effects, such as
brain-swelling, and the potential cost.
"Aducanumab's efficacy as a treatment for the cognitive dysfunction in
Alzheimer's disease cannot be proven by clinical trials with divergent
outcomes," said Mayo Clinic neurologist Dr. David Knopman, who was recused from
the expert panel because he helped conduct the trials.
Others believe the FDA could approve the drug without another trial.
Previous clinical studies had largely targeted patients in later stages of the
disease, while many experts now believe attacking Alzheimer's as early as
possible may be the key to success. But there was a lack of clarity on how to
assess a drug's success when functional deficits are less pronounced.
In 2018, the FDA revised its standard of proof guidance for reviewing
Alzheimer’s drugs by essentially combining what had been separate goals for
cognition, or memory, and day-to-day function. The new guidance stressed the
need for a drug to show “clinically meaningful” benefits, a term it has not
clearly defined.
Alzheimer's advocacy groups are pushing for a broad definition, saying it should
include preserving the ability to perform daily activities such as shopping
independently or remembering to turn off a stove.
“They have been trying to lower the bar and help any company to get a drug
approved,” said Dr. Marwan Sabbagh, from the Cleveland Clinic Lou Ruvo Center
for Brain Health in Las Vegas. "How much (improvement) do you need to be
clinically meaningful? Naturally this is very subjective.”
The FDA did not immediately respond to a request for comment.
(Reporting by Deena Beasley; Editing by Caroline Humer and Bill Berkrot)
[© 2020 Thomson Reuters. All rights
reserved.] Copyright 2020 Reuters. All rights reserved. This material may not be published,
broadcast, rewritten or redistributed.
Thompson Reuters is solely responsible for this content. |
