FDA approves first drug with hope for Alzheimer's

A controversial drug from Cambridge-based Biogen that aims to slow the progression of the deadly disease in people with early symptoms.

Seven months after an advisory panel of medical professionals concluded that Biogen did not provide enough evidence to recommend approval, the Food and Drug Administration rejected that recommendation and approved the drug, called aducanumab. When launched, it will enter under the Aduhelm brand and is expected to generate billions of dollars in annual revenue. The company was not told how much it would cost.

But FDA approval required Biogen to run a larger clinical trial that showed that its ability to remove a substance called amyloid, which builds up in the brains of Alzheimer's patients, has cognitive benefits. Without this approval, Biogen will eventually have to stop marketing the drug.

Director of the FDA Center for Drug Evaluation and Research, Dr. "Aduhelm is the first new treatment approved for Alzheimer's disease since 2003," Patricia Cavazzoni said in a statement. "Perhaps more importantly, Aduhelm is the first treatment to address the underlying pathophysiology of Alzheimer's disease, the presence of amyloid beta plaques in the brain."

The decision will be applauded by Alzheimer's patients, their families and advocates, who are hungry for a drug more effective than the five previously approved drugs. Alzheimer's affects an estimated 5.8 million Americans and is the sixth leading cause of death in the United States.

It was also a victory for Biogen, which has the largest headcount of any domestic pharmaceutical company in Massachusetts; About 2,400 of its 9,100 employees worldwide work in the state.

“This historic moment is the culmination of more than a decade of groundbreaking research in the complex field of Alzheimer's disease,” said Michel Vounatsos, CEO of Biogen. We believe this world-class drug will transform the treatment of people living with Alzheimer's disease and trigger continuous innovation for years to come.”

Specializing in the treatment of neurological disorders such as multiple sclerosis, Biogen had made aducanumab a key component in its long-term business strategy. The biotech added 1,700 employees to its global workforce in 2020, at least in part in anticipation of aducanumab approval. It also established a $1 billion facility in Switzerland to assist in the manufacture of the drug.

The drug is designed to treat patients in the early stages of the disease, and some experts estimate that about 2 million people in the United States may be eligible to take the drug.

Maria Carrillo, chief science officer for the Alzheimer's Association, a Chicago-based advocacy group, said recently that aducanumab isn't perfect, but families are desperate for a treatment that will keep their loved ones' minds intact longer early in the disease.

The association commissioned celebrities such as Samuel L. Jackson to lobby for approval in a campaign called "More Time." Jackson lost several members of his family to the disease, including his mother, who died in 2012.

Some, however, opposed approval. They said they wouldn't prescribe the drug because they weren't convinced it worked, given the conflicting results of two late-stage clinical trials.

Also, aducanumab is not without risk for patients. Three out of 10 patients who received high doses in clinical studies had brain swelling detectable on an MRI scan. This did not usually cause serious symptoms, but did cause confusion and falls in some patients.

Aducanumab is a monoclonal antibody made from immune cells of elderly people with little or no cognitive decline. The researchers designed it to remove a sticky substance called beta amyloid that builds up in the brains of Alzheimer's patients. Research shows it does this well. But it's unclear whether reducing amyloid plaques, whose role in Alzheimer's has been fiercely debated for decades, slows the disease process that kills brain cells and affects memory and thinking.

The drug's adventure has been marked by rising hopes and discouraging setbacks that have rattled Biogen's stock valuation by billions of dollars in recent years. After aducanumab showed promise in 2015, Biogen launched two late-stage trials, but they were discontinued in March 2019 after an independent monitoring panel concluded that the drug didn't work.

In a stunning comeback in October 2019, Biogen announced that analysis of additional data has led to a resurgence of the drug. In one of the trials, people given aducanumab fell 22 percent slower than those given a placebo. The other trial failed. Still, Biogen concluded that aducanumab slows cognitive decline and that the key to effectiveness is to give patients high enough doses.

The FDA typically requires two convincing studies for a drug to be approved, but has made exceptions, especially for deadly or devastating diseases for which there are no effective treatments. Biogen, a Japanese drug on aducanumab.

https://www.bostonglobe.com/2021/06/07/business/fda-oks-biogens-alzheimers-drug-first-new-medicine-disease-18-years/