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- The US Food and Drug Administration (FDA) approves the first new drug for the treatment of Alzheimer’s in almost two decades
- Known as aducanumab and sold under the brand name Aduhelm, the drug is the first ever to treat the disease itself rather than symptoms of dementia
- However, many medical experts and FDA advisors claim there is not enough evidence to show the drug’s ability to slow cognitive decline in patients
- Under the terms of the FDA approval, developer Biogen will now need to conduct a phase four clinical trial for the drug
- If the results aren’t strong enough to prove the drug’s ability to slow cognitive decline in Alzheimer’s patients, the FDA has the right to rescind its approval
United States drug regulators have this week approved the first new drug for the treatment of Alzheimer’s disease in almost two decades.
The decision came despite the Food and Drug Administration (FDA) facing opposition from several advisors and experts who said there was not enough evidence to show the drug can help patients.
Developed by American biotech giant Biogen alongside Japanese pharmaceutical company Eisai, the drug is the first-ever medication aimed at treating Alzheimer’s disease itself rather than simply treating the symptoms associated with dementia.
Known as aducanumab, the drug will be sold under the brand name Aduhelm.
Biogen CEO Michael Vounatsos said he was “incredibly humbled” by the FDA approval for the drug.
“We come to this historic moment with thanks for the inspiring contributions of thousands of Alzheimer’s patients and caregivers who participated in our clinical trials, for the pioneering work of scientists and researchers inside and outside Biogen, and for the relentless dedication of our employees globally,” Mr Vounatsos said.
However, the FDA approval for the drug was not met with as much enthusiasm from other experts in the industry.
Dr Caleb Alexander, an FDA adviser and researcher at John Hopkins University, said he was “surprised and disappointed” by the FDA’s decision.
“The FDA gets the respect that it does because it hs regulatory standards that are based on firm evidence,” Dr Alexander, who recommended against the drug’s approval, said.
“In this case, I think they gave the product a pass.”
Why the opposition?
Aduhelm works by reducing the presence of a protein called amyloid-beta in the brain with the intent of slowing the process of cognitive decline in Alzheimer’s patients. The drug has not been shown to reverse the process.
The controversy around the drug comes from two phase-three trials for Aduhelm with contradicting results: one trial suggested the drug can delay cognitive decline by around 22 per cent, while the other suggested it has no benefit.
In fact, both trials were abandoned in March 2019 when independent monitors found the drug was unlikely to benefit patients. It was only later that year that Biogen released a new analysis of one of the studies suggesting the drug could slow cognitive decline.
As such, many trial researchers have come forward and said they believe there is not enough evidence to warrant the FDA approval.
Conditional approval
Despite the concerns from advisors, the FDA decided Aduhelm meets the standards for “accelerated approval”, which is a type of approval used to fast-track new drugs being developed to treat serious conditions.
However, the FDA approval comes a string of conditions. Among these is the requirement for Biogen to conduct a new clinical trial for the drug.
Under this phase four trial, Biogen will work to provide more evidence of the drug’s effectiveness. If the drug is proven ineffective, the FDA has the right — but not the obligation — to pull Aduhelm from shelves and rescind its approval of the drug.
Aduhelm is administered as a monthly intravenous infusion, with Biogen pricing the drug at US$56,000 (around A$72,000) for a year’s worth of treatment. The majority of patients won’t pay anywhere near the full amount, however, thanks to insurance coverage and other discounts.
Around six million people in the United States and 30 million people across the world suffer from Alzheimer’s disease.
