Controversial drug for Alzheimer's wins approval from FDA

The US Food and Drug Administration (FDA) approved the first new medication for Alzheimer's disease in nearly 20 years on Monday even though independent advisers said that the treatment hasn't been shown to help slow the brain-destroying disease.

The fast-track authorization by the FDA means Biogen, which developed the drug with Eisai Co of Japan, can begin distributing Aduhelm, which has the scientific name aducanumab.

Aduhelm received attention prior to its approval because experts disagreed on the drug's effectiveness, and some expressed concern about side effects.

Dr Caleb Alexander, an FDA adviser who recommended against the drug's approval, said 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," Alexander, a researcher at Johns Hopkins University, told The Associated Press.

Others hailed the FDA action.

"Today's approval of Aduhelm is a transformational breakthrough in the fight to stop this horrible disease. After years of disappointment and despair, this decision offers new hope for many families and a trigger for future investment and innovation," said George Vradenburg, chairman and co-founder of Us Against Alzheimer's in Biogen's statement.

Dr Stephen Salloway, director of Neurology and the Memory and Aging Program at Butler Hospital in Indiana, said, "This approval represents a major advance in the treatment of Alzheimer's disease."

Dr Patrizia Cavazzoni, director of the FDA's Center for Drug Evaluation and Research, acknowledged the debate over the drug in the agency's announcement on Monday.

"There has been considerable public debate on whether Aduhelm should be approved. As is often the case when it comes to interpreting scientific data, the expert community has offered differing perspectives," she said.

The drug was approved using the Accelerated Approval pathway under which there still remains some uncertainty about the drug's clinical benefit despite that the drug may provide meaningful therapeutic benefit over existing treatments.

Aducanumab can reduce the level of amyloid plaques in the brain, and therefore is expected to lead to a reduction in the clinical decline of dementia, according to the FDA.

This approval is significant because, besides being the first novel therapy approved for Alzheimer's disease since 2003, Aduhelm is the first treatment directed at the underlying pathophysiology of Alzheimer's disease, the presence of amyloid beta plaques in the brain.

"This historic moment is the culmination of more than a decade of groundbreaking research in the complex field of Alzheimer's disease. 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," Michel Vounatsos, CEO at Biogen, said in a statement.

In the US, roughly 6.2 million people aged 65 and above have Alzheimer's dementia, according to the 2021 data released by the Alzheimer's Association. That number is projected to double to 12.7 million by 2050. About 2 million people in the US have mild Alzheimer's-related dementia that Aducanumab can be used for, according to Biogen.

Aware of the different views on the drug from the medical field, FDA said it had examined the clinical trial data, solicited input from the Peripheral and Central Nervous System Drugs Advisory Committee, listened to the patients, and granted the conditional approval by requesting the drug companies to conduct post-approval studies to verify the anticipated clinical benefit. If the studies don't verify its anticipated clinical benefit, the FDA could remove the drug from the market.

The medication will be given as an infusion every four weeks. Biogen didn't immediately disclose the price, though the AP said analysts have estimated the drug could cost between $30,000 and $50,000 for a year's worth of treatment.

A preliminary analysis by one group found that the drug would need to be priced at $2,500 to $8,300 per year to be a good value based on the "small overall health gains" suggested by company studies, AP said.

Biogen's stock was halted for the announcement. It closed up 38 percent at $395.85.

Jing Shiyan in Kansas contributed to the story.