FDA approves first new Alzheimer’s drug in two decades

pharmafile | June 8, 2021 | News story | Manufacturing and Production  

Biogen’s controversial new drug, aducanumab, has been accepted by the FDA, making it the first Alzheimer’s drug to be approved in nearly 20 years.

Though this is a huge step for the disease, as no new drug has been approved in the past 18 years despite more than 400 clinical trials and billions of dollars being spent, the effectiveness of aducanumab is disputed by scientists.

Aducanumab targets the underlying cause of Alzheimer’s, the most common form of dementia, rather than its symptoms.

The FDA said the accelerated approval is “based on the drug’s effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients”.

But many scientists are conflicted over whether the drug actually works.

In March 2019, late-stage international trials of aducanumab, involving about 3,000 patients, were halted when analysis showed the drug, given as a monthly infusion, was not better at slowing the deterioration of memory and thinking problems than placebo.

But later that year, Biogen analysed more data and concluded the drug did work, as long as it was given in higher doses. The company also said it significantly slowed cognitive decline.

Due to this uncertainty, the FDA said it is “requiring the company, Biogen, to conduct a new randomised, controlled clinical trial to verify the drug’s clinical benefit. If the trial fails to verify clinical benefit, the FDA may initiate proceedings to withdraw approval of the drug.”

The approval has led charities, such as Alzheimer’s Research UK, to call on the government to prioritise and accelerate research into aducanumab, where at least 100,000 people who suffer from of the disease would be eligible for the drug.

Some experts emphasise what this breakthrough will mean for people who are affected by Alzheimer’s, Dr Gen Li, Founder and President at Phesi, said: “Beyond the immediate implications for Biogen, what this debate around aducanumab has underscored is that neurodegenerative diseases like Alzheimer’s remain an incredibly emotive and fraught subject.

“For Biogen, it might have been a ‘bet the farm’ approach, but for sufferers, their families and their carers, even the slightest possibility of a treatment naturally holds much hope.

“The landmark FDA decision made today will be discussed at length, but it’s important to remember that neurodegenerative disease is one of the biggest unmet needs humanity faces and continued R&D is imperative.”

Others feel that the data is not strong enough to justify the approval, Maxim Jacobs, Managing Partner and Director of Research at Edison Group, said: “Today’s approval of Aduhelm (aducanumab) from Biogen is being heralded as a historic event for Alzheimer’s. 

“However, the data has been inconsistent and if this were a data package for another disease, I’m not sure it would have been approved.  There was likely quite a bit of political pressure put on the FDA to approve the drug, both from the Biden administration, which recently made strong comments about the need to have more treatments for the disease a couple of week ago, as well as from other government officials (Biogen significantly increased their lobbying spending in 2020/2021). 

“As a result, Biogen will receive a windfall of billions of dollars in annual revenues for a drug that may not actually help much.”

Robert Howard, Professor of Old Age Psychiatry at UCL, went further, he said: “As a dementia clinician and researcher with personal family experience of Alzheimer’s disease, I want to see effective dementia treatments as much as anyone.

“I consider the approval of aducanumab represents a grave error that will have only negative impact on patients and their families and that could derail the ongoing search for meaningful dementia treatments for a decade.

“Amazingly, the FDA have sidestepped available clinical trial outcomes data that indicate the drug probably doesn’t work.”

If the drug were to be approved in the UK, there would be many things to consider. Patients with very early memory problems will need PET scans of the brain to establish whether they have amyloid plaques. This would require expensive equipment and trained staff.

There would likely be considerable debate on the cost effectiveness of the drug as an NHS resource.

The WHO estimates that 50 million people around the world suffer with dementia, with Alzheimer’s contributing up to 70% of those cases. 10 million new cases are added every year.

Lilly Subbotin

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