FDA faced with tough decision in approving Alzheimer’s drug

The Food and Drug Administration is on the verge of announcing one of its most contentious decisions in years: the fate of an Alzheimer’s drug that could be the first treatment approved after nearly two decades of failed efforts to find ways to curb the debilitating disease.

On Monday, the agency will rule on the drug, aducanumab, which aims to slow progression of memory and thinking problems early in the disease. If approved, it would be the first new Alzheimer’s medication since 2003 and the first treatment on the market that attacks the disease process rather than just easing symptoms.

It would become a blockbuster drug within several years, analysts predict, costing tens of thousands of dollars annually per patient and bringing a windfall to its manufacturer, Biogen.

Patient groups, desperate for treatments, are pushing for approval. But greenlighting the drug would fly in the face of objections from several prominent Alzheimer’s experts and the FDA’s independent advisory committee.

In November, the committee voted overwhelmingly against recommending approval, saying data failed to demonstrate persuasively that aducanumab slowed cognitive decline. Other experts say aducanumab had not shown convincing benefit to outweigh its safety risks.

About 6 million people in the United States and roughly 30 million globally have Alzheimer’s, a number expected to double by 2050. Currently, five medications approved in the United States can delay cognitive decline for several months in various Alzheimer’s stages. About 2 million Americans have mild Alzheimer’s-related impairment, fitting criteria for aducanumab, a monthly intravenous infusion requiring regular imaging to detect potential brain swelling.

Biogen officials declined to comment, but in earnings calls, medical meetings and FDA presentations, they have said the evidence shows cognitive benefit.

Debate centers on two never fully completed phase three trials that contradicted each other. One suggested that a high dose could slightly slow cognitive decline; the other showed no benefit. Biogen says that given the need for Alzheimer’s medications, the single positive trial, plus results from a small safety trial and aducanumab’s ability to reduce a key protein, should justify approval.

The FDA typically follows advisory committee recommendations and usually requires two convincing studies for approval, but it has made exceptions, especially for severe diseases that lack treatments.

Two other medications now in trials appear more promising than aducanumab, but it could be three or four years before data would indicate whether they merit approval.