11:21 a.m. ET June 7, 2021: From the Associated Press -- The FDA has 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 FDA is requiring the drugmaker to conduct a follow-up study to confirm the drug’s 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.
On Monday, the U.S. Food and Drug Administration is expected to announce whether a new drug to treat Alzheimer’s disease will be approved for use in the United States.
The drug, made by Biogen Inc. and called aducanumab, would be the first treatment in decades to be approved for use by patients of the mind-wasting disease.
However, possible approval of aducanumab has sparked debate. Those who want to see the drug approved for use point to the fact that the treatment is the first designed to slow the course of Alzheimer’s disease rather than treat symptoms of the disease.
Those who oppose the approval of the drug point to its cost, its safety record and the question of whether the drug can provide meaningful benefit to a patient.
Aducanumab is a monoclonal antibody engineered in a laboratory. It is designed to stick to the beta-amyloid molecule that forms plaques in the brains of people with Alzheimer’s. Studies have indicated that the drug does do that.
The predominant theory of what causes Alzheimer’s is that an abnormal accumulation of beta-amyloid and another abnormal protein, tau, causes the memory and function issues associated with the disease.
According to Biogen, once aducanumab sticks to the plaque, the body’s immune system would see the plaque as a foreign invader and would launch an attack to kill it.
The drug’s developer says it hopes that brain cells will stop dying once the plaques are gone, and thinking, memory, function and behavior will stop deteriorating.
According to available data, there was one phase two clinical trial to assess efficacy and side effects, and two phase three clinical trials to assess the safety of the drug in addition to the effectiveness and side effects.
The phase two study and one of the phase three studies showed promise, meaning that the drug worked to slow down the decline of Alzheimer’s. The other phase three study showed the drug was not effective.
Side effects from the drug included bleeding in the brain, the company reported.
That study led the company to scrap the work on the drug in March 2019. In October 2019, Biogen said a more detailed company analysis showed some patients with very early forms of the disease benefited from taking higher doses over an extended period of time, Reuters reported.
In November 2020, the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee rejected the data presented about aducanumab, saying the positive results were not representative of the drug.
The committee voted 10-0 against the drug with one member abstaining.
Another concerning aspect of the drug is the cost – it has a projected price tag of about $50,000 per year per patient.
A rejection of the drug would likely mean the end of aducanumab and possibly the end of research on similar Alzheimer’s drugs in development, RBC Capital Markets analyst Brian Abrahams said.
It is “likely to reverberate throughout the biopharma sector, influencing overall sentiment on the space, perceptions on regulatory flexibility, and business development dynamics,” Abrahams told Barrons.
Approval for the use of the drug could mean options in the treatment of the disease that patients do not have now, Harry Johns, president and CEO of the Alzheimer’s Association said.
“We believe strongly that if the FDA approves this treatment, it will be a new day for Alzheimer’s.”
The Alzheimer’s Association backs approval of aducanumab even with some trials showing a modest ability to slow down the disease.
“This is not a cure,” Johns says. “This is an incremental benefit, potentially, and that benefit can be very real in changing lives for so many.”
Nearly six million people in the United States and roughly 30 million around the world have Alzheimer’s disease. That number is expected to more than double by 2050. Alzheimer’s disease is the sixth leading cause of death in the United States.
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