Story at a glance
- A lack of strong data from clinical trials casts doubt over the new Alzheimer drug’s efficacy, the Wall Street Journal reports.
- The manufacturer Biogen said more clinical studies focused on “high-quality data” are underway.
The medication designed to treat Alzheimer’s disease and that was approved by the U.S. Food and Drug Administration (FDA) as the first treatment to get the regulatory green light in more than two decades received pushback from medical professionals and, apparently, from fellow public health regulators.
New internal reports obtained and reviewed by The Wall Street Journal suggest that FDA officials debated granting approval for the drug, from pharmaceutical company Biogen, following objections from the statistics division within the agency.
Disputes were broadly in regards to whether the evidence from clinical trials was strong enough to support the theory that Biogen’s drug helped slow the spread of Alzheimer’s.
“FDA, then, is faced with a situation where the available evidence on the clinical endpoints is short of what we would require for standard approval of aducanumab,” Peter Stein, the director of the FDA’s office of new drugs, reportedly wrote in one of the obtained memos. “At the same time, patients with [Alzheimer’s] have a serious, progressive, ultimately fatal disease and are desperate for treatments.”
FDA Acting Commissioner Janet Woodcock acknowledged that while there are different opinions about the agency’s decision, “an experienced group of medical reviewers” concluded the drug warranted approval.
Some of the criticism of the drug, called Aduhelm, comes from its focus on beta amyloid plaque buildup in the brain. Several doctors and scientists say that there isn’t enough evidence to suggest that targeting this substance helps alleviate Alzheimer’s.
The disease is highly prevalent among older U.S. adults, with an estimated 6 million cases in the country. Broad demand for a treatment could have influenced the FDA’s approval decision.
“There remains residual uncertainty regarding clinical benefit,” Stein noted, adding that if the FDA waited for results from another study, current patients “could suffer irreversible loss of brain neurons and cognitive function and memory.”
Biogen released its own statement in light of the speculation surrounding its plans for Anduhelm, saying that the company has been working with private and public health care payers to negotiate a price for the medication.
It also added that the company is working on conducting an additional clinical trial to confirm results.
“We are working with urgency and putting resources and plans in place towards the goal of completing the confirmatory trial ahead of the nine-year timeframe, with a focus on high quality data,” officials wrote.