FDA considering accelerated approval of new Alzheimer’s drug

The FDA is considering accelerated approval of a new drug shown to attack amyloid plaque in the human brain, a contributing cause of cognitive dysfunction in dementia and Alzheimer’s disease.  The FDA is expected to reach a decision by Friday, January 6.

"Accelerated approval means that they believe the drug does what it is supposed to do, namely removing amyloid from the brain. But they’re not totally convinced that it has clinical impact," said Dr. Ronald Petersen, an Alzheimer’s researcher with Mayo Clinic in Rochester, Minnesota.  He explained the accelerated approval process means the drugmaker has to conduct more research to show that removing amyloid plaque has a clinical benefit to patients.

The drug is called lecanemab. In a Phase 3 trial released this fall of 1,795 people with mild cognitive impairment and mild dementia, the drug’s makers Eisai and Biogen reported lecanemab appeared to show a 26 percent reduction of cognitive decline after 18 months of treatment.

Mayo Clinic did not participate in the trial, and Dr. Petersen does not conduct any paid research for Eisai.  He says these latest findings are not part of the drugmakers' formal application to the FDA for accelerated approval.  But he believes the trial showed the drug is effective in lowering amyloid levels in the brain.

"In fact, two-thirds of the people in the study actually had their amyloid levels lowered to normal or at the threshold of normality," explained Petersen. "From a clinical perspective, it slowed down the progression of the disease.  It didn’t stop the disease, it didn’t make people better, but it slowed down the rate at which the disease progresses."

But the trial also raised safety concerns with two side effects of brain swelling and bleeding.

"In many people with Alzheimer’s disease, they not only have amyloid protein in the brain, but they also have it in the blood vessels," said Petersen. "So when the drug removes amyloid from the brain, it also removes it from the blood vessels, making them leaky. So this can lead to swelling and can lead to bleeding in the brain."

Aside from more research by Eisai and Biogen if the FDA grants accelerated approval, the other factors yet to be decided are price and coverage.  The makers have not yet set a price for the drug, and organizations such as the Centers for Medicare and Medicaid Services have not yet decided to cover the drug.

After the results of the Phase 3 trial were published in the New England Journal of Medicine in November, the Alzheimer’s Association called on the FDA to grant its accelerated approval.

"These peer-reviewed, published results show lecanemab will provide patients more time to participate in daily life and live independently," said the Alzheimer’s Association in a statement.

The association projects the disease will affect 120,000 Minnesotans by 2025, an increase of 21% in just five years.