Top FDA Staffers Defend Approval of Biogen’s Alzheimer’s Drug in Medical Journal
[Top FDA Staffers Defend Approval of Biogen’s Alzheimer’s Drug in Medical Journal - MarketWatch](https://www.marketwatch.com/articles/top-fda-staffers-defend-approval-of-biogens-alzheimers-drug-in-medical-journal-51626265032?mod=barrons-on-marketwatch)
Published: July 14, 2021 at 8:17 a.m. ET
By [Josh Nathan-Kazis](https://www.barrons.com/authors/8583_BARRONS?mod=article_byline)
Referenced Symbols: [**BIIB+0.83%** ](https://www.marketwatch.com/investing/stock/BIIB?mod=refsymb_bomw)
Top U.S. Food and Drug Administration officials who signed off on the approval of [Biogen](https://www.marketwatch.com/investing/stock/BIIB?mod=MW_story_quote)’s Alzheimer’s disease therapy last month defended their decision in a medical journal article published Tuesday, [writing that patient representatives](https://jamanetwork.com/journals/jamainternalmedicine/fullarticle/2782119#related-articles-tab) had told them they were willing to accept uncertainty about the drug’s clinical benefit in return for quicker access to the treatment.
“If instead, approval had been delayed, the loss of brain function in these patients over this time—which might have been lessened by earlier initiation of treatment—would not be regained,” wrote the three FDA leaders, including Dr. Patrizia Cavazzoni, who leads the FDA division responsible for the approval of most new drugs.
The letter comes amid a storm of condemnation for the agency over its approval of the [Biogen](https://www.marketwatch.com/investing/stock/BIIB?mod=MW_story_quote) (ticker: BIIB) drug, known as Aduhelm, which has not been proven to be effective at slowing patients’ clinical decline, and which will cost $56,000 a year.
Three members of [an FDA advisory committee resigned over the approval, ](https://www.marketwatch.com/articles/fda-biogen-alzheimers-drug-51625847246)and the FDA’s acting commissioner has [asked for an investigation of interactions between FDA staff and Biogen.](https://www.marketwatch.com/articles/biogen-stock-alzheimer-fda-investigation-51626098930)
As *Barron’s* reported last weekend, the approval has led some scientists to warn that the FDA may be [de-emphasizing its role as a gatekeeper between the biopharma industry and the public.](https://www.marketwatch.com/articles/fda-biogen-alzheimers-drug-51625847246)
In their joint article published in Tuesday in the medical journal JAMA Internal Medicine, however, the three FDA officials most closely associated with the decision framed the approval as a response to an “urgent need” for Alzheimer’s disease therapies.
No new Alzheimer’s disease treatment had been approved in nearly two decades when the agency approved Biogen’s drug.
The FDA officials wrote that they agreed that Biogen’s trials of Aduhelm had not proven it had a clinical benefit. “After reaching this conclusion, we faced a challenge: available evidence was strongly suggestive of a benefit from aducanumab but was complicated and in some respects contradictory; yet patients with Alzheimer’s disease are in urgent need of treatments that delay disease progression and loss of function,” they wrote.
As such, they say they decided to support accelerated approval for Aduhelm, a regulatory pathway often used to approve cancer drugs that have been shown to have an effect on markers of disease, but for which clinical benefits take too long to measure.
“In weighing the benefits vs the risks of aducanumab, we considered that Alzheimer’s disease leads to irreversible loss of memory, cognition, and ability to function in daily activities,” the authors wrote. “In listening sessions, we heard from patients and their families about the devastating toll the disease has taken and their desire for a treatment to stop or delay functional losses.
In addition to Cavazzoni, director of the Center for Drug Evaluation and Research at the FDA, the paper’s other authors are Dr. Peter Stein, who leads the Office of New Drugs within CEDR, and Dr. Billy Dunn, director of the Office of Neuroscience, which is the subsidiary of the Office of New Drugs that is responsible for Alzheimer’s therapies, among other medicines.
The health news website STAT [reported in late June](https://www.statnews.com/2021/06/29/biogen-fda-alzheimers-drug-approval-aduhelm-project-onyx/) that Dunn had sat down with Biogen’s “off-the-books meeting” with Dunn in May 2019 after Biogen had stopped two studies of Aduhelm on the recommendation of an independent data monitoring committee, and before the company had officially told the FDA that a new analysis of the data suggested the drug might work after all.
The letter from FDA acting director Dr. Janet Woodcock asking for an investigation into the Biogen approval refers indirectly to questions raised [by the STAT story.](https://www.marketwatch.com/articles/fda-investigation-biogen-alzheimers-drug-51625856966)
JAMA Internal Medicine also published dissenting articles on Tuesday, including one wrote by one of the members of the advisory committee who resigned following the approval, Dr. Aaron Kessehleim, a professor of medicine at Harvard Medical School. Kesselheim and two co-authors argued in their piece that the accelerated approval pathway itself needs reform.
“For the public and physicians to trust the pathway and the clinical value of drugs with accelerated approval, reforms as well as the timely completion of post-approval trials are needed,” [Kesselehim wrote.](https://jamanetwork.com/journals/jamainternalmedicine/fullarticle/2782120)
Biogen shares were up 0.8% on Tuesday, and flat in pre-market trading Wednesday. The stock is up 43.7% so far this year.
Write to [josh.nathan-kazis@barrons.com](mailto:editors@barrons.com)
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