Combative U.S. FDA panel votes against Biogen Alzheimer's drug

Washington

A panel of outside advisers to the U.S. Food and Drug Administration on Friday voted that a potential Alzheimer’s treatment from Biogen Inc has not been proven to slow progression of the disease, a sharp rebuke to agency staff who earlier this week praised the drug.

The FDA still could decide to approve the drug, aducanumab, making it the first new Alzheimer’s treatment in decades. But the group of scientific advisers made their doubts about the treatment clear.

Several of the 11 panel members expressed resentment that the FDA’s early view of aducanumab - which was made public on Wednesday - was biased toward a favorable outcome for Biogen. Those FDA staff comments sent Biogen shares soaring. They were halted during Friday’s panel meeting.

The agency did “an extraordinary amount of explaining around contrary findings,” said Dr. Caleb Alexander, professor of epidemiology and medicine at Johns Hopkins Bloomberg School of Public Health.

The panel voted “no” to three questions related to whether a single successful large trial of aducanumab was enough evidence of the drug’s effectiveness given the clear failure of a second large study. They questioned the FDA’s stance that an early-stage trial of the drug was supportive enough to offset the failure of the second pivotal trial.

Five panel members did vote “yes” to the question of whether aducanumab has an effect on levels of amyloid plaque in the brains of people diagnosed with early Alzheimer’s, but six voted “uncertain” on the question.

Trial data - detailed by Biogen and the FDA at the meeting - included lots of “red flags,” said committee member Dr. Aaron Kesselheim, associate professor at Harvard Medical School.

“We will continue to work with the FDA as it completes its review of our application,” Biogen said in a statement.

The FDA is expected to make its decision on the drug by March and is not obligated to follow the recommendations of the panel.

Alzheimer’s disease is the sixth leading cause of death in the United States and there is “a profound and enormous unmet medical need” for new treatments, Billy Dunn, director of neurology products for the FDA’s Center for Drug Evaluation and Research, said during the meeting.

The advisory committee on Friday also heard testimony from a number of people suffering from Alzheimer’s, including several who said they had benefited from aducanumab while enrolled in trials of the drug, adding pressure on the agency to approve it.

“We know firsthand that it was helping Kevin,” said Kim Bonham, speaking of her husband, who was diagnosed with Alzheimer’s in 2016.

Biogen in October last year revived its plans to seek approval for aducanumab, months after it had said that an independent futility analysis of its two pivotal trials showed they were unlikely to succeed.

The company said it changed course after a new analysis showed a high dose of the drug could slow disease progression.

The FDA staff earlier this week said data from one of the trials “provides the primary evidence of effectiveness” and a clinically meaningful treatment effect.

They said failure of a second large trial did not detract from the findings of the positive study.

“FDA is gate-keeper and believes in the totality and safety is OK, so we think they could still approve,” said Jefferies analyst Michael Yee.