United States health officials have approved a controversial new drug for the treatment of Alzheimer’s disease, the first new medication for the disease in 18 years.
The US Food and Drug Administration (FDA) confirmed its approval for the treatment on Monday (US time) despite opposition from its independent advisory committee and some Alzheimer’s experts that there was not enough evidence that the drug could help patients.
The drug, called aducanumab but marketed as Aduhelm by pharmaceutical company Biogen, received approval under the FDA’s accelerated approval pathway, which provides patients suffering from serious disease earlier access to drugs when there is an expectation of clinical benefit despite uncertainty about its benefits.
The drug is currently under review by the Therapeutic Goods Administration (TGA) in Australia with an announcement expected in early 2022.
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Dr Patrizia Cavazzoni, from the FDA’s Center for Drug Evaluation and Research, said this treatment was different from other Alzheimer’s drugs in that it didn’t just attempt to treat the symptoms of the disease.
“Alzheimer’s disease is a devastating illness that can have a profound impact on the lives of people diagnosed with the disease as well as their loved ones,” she said.
“Currently available therapies only treat symptoms of the disease; this treatment option is the first therapy to target and affect the underlying disease process of Alzheimer’s.
“As we have learned from the fight against cancer, the accelerated approval pathway can bring therapies to patients faster while spurring more research and innovation.”
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Dementia Australia chief executive Maree McCabe welcomed the FDA announcement and said it was exciting news for the entire dementia community.
“This announcement provides real hope for people who are developing symptoms and in the early stages of Alzheimer’s disease,” Ms McCabe said.
“Importantly this will result in further research investment into more treatments for all forms of dementia.”
Alzheimer’s is an irreversible, progressive brain disorder that slowly destroys memory and thinking skills, and eventually the ability to carry out simple tasks.
While the specific causes of Alzheimer’s disease are not fully known, it is characterised by changes in the brain – including amyloid plaques – that result in loss of neurons and their connections. These changes affect a person’s ability to remember and think.
Aduhelm represents a first-of-its-kind treatment approved for Alzheimer’s disease, according to the FDA.
It is the first new treatment approved for Alzheimer’s since 2003 and is the first therapy that targets the fundamental pathophysiology of the disease.
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Researchers evaluated Aduhelm’s efficacy in three separate studies representing a total of 3482 patients with Alzheimer’s disease.
Patients receiving the treatment had significant dose and time-dependent reduction of amyloid beta plaque, while patients in the control arm of the studies had no reduction of amyloid beta plaque.
The approval for the treatment, which needs to be given as an infusion every four weeks, requires Biogen to verify its clinical benefits in a post-approval trial. If it cannot verify these benefits, the FDA may initiate proceedings to withdraw approval for the drug.
Biogen chief executive Michel Vounatsos said the approval would change the lives of many people suffering with Alzheimer’s disease.
“This historic moment is the culmination of more than a decade of ground-breaking research in the complex field of Alzheimer’s disease,” he said.
“We believe this first-in-class medicine will transform the treatment of people living with Alzheimer’s disease and spark continuous innovation in the years to come.
“Together with the healthcare community, we are ready to bring this new medicine to patients and begin to address this growing global health crisis.”
Some doctors have expressed concerns over the drug’s effectiveness.
Dr Caleb Alexander, who was a member of the FDA advisory committee and advised against approval, told the ABC that 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,” he said.
Dr Lon Schneider, from the California Alzheimer’s Disease Center, who helped conduct one of the aducanumab trials, told The New York Times that he also questioned whether the treatment would be effective.
“There’s so little evidence for effectiveness,” Dr Schneider said. “I don’t know what caught the FDA’s fancy here.”
As YourLifeChoices reported in November, the treatment received a scathing assessment from the FDA’s panel of neurological experts, including Dr Alexander.
Are you worried that the FDA approval for the Alzheimer’s drug treatment will give false hope to sufferers? Or are you glad to see a new drug on the market? Why not share your thoughts in the comments section below?
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