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Biogen defends Aduhelm approval process, says ‘misinformation’ is fueling controversy

Aduhelm, being administered above, has a $56,000 annual price tag, and critics have decried the process by which it was approved.
Aduhelm, being administered above, has a $56,000 annual price tag, and critics have decried the process by which it was approved.Jessica Rinaldi/Globe Staff

Biogen on Thursday vigorously defended its actions that led to the approval of its controversial new Alzheimer’s drug and said negative media coverage was misleading patients who might benefit from the medicine.

Michel Vounatsos, chief executive of the Cambridge biotech, said Biogen stands behind the clinical trial data that led to the June 7 approval of Aduhelm and that there was nothing unusual about interactions between company employees and federal regulators.

Vounatsos, who has run Biogen since 2017, blamed media coverage for the controversy.

“But we are not the ones suffering the most,” he said at a quarterly earnings presentation, in some of his lengthiest public remarks since Aduhelm’s approval. “Whatever the motives of the controversy are, the ones who are potentially misled, confused, denied help are the patients.”

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The approval by the US Food and Drug Administration has triggered an extraordinary backlash from some doctors, insurers, and advocates for patients. Aduhelm, which generated conflicting results in two late-stage trials, has a $56,000 annual price tag, and critics have decried the process by which it was approved.

Few patients appear to have received the commercial drug, which has generated about $2 million in sales so far. However, the federal Medicare program has only begun its review for coverage of the medicine, which is widely expected to be a blockbuster, defined as sales of $1 billion or more.

News reports by STAT and The New York Times have depicted an unusually cozy relationship between Biogen executives and regulators before the FDA greenlighted Aduhelm, including previously undisclosed meetings to discuss a pathway for approval.

The FDA’s acting commissioner, Dr. Janet Woodcock, on July 9 asked the Department of Health and Human Services’ independent Office of the Inspector General to investigate the process that led to the approval. She cited interactions between Biogen employees and drug regulators, first reported by STAT, that “may have occurred outside of the formal correspondence process.”

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Aduhelm, Biogen's drug for Alzheimer's disease, at Butler Hospital in Rhode Island.
Aduhelm, Biogen's drug for Alzheimer's disease, at Butler Hospital in Rhode Island.Jessica Rinaldi/Globe Staff

Before the earnings presentation Thursday morning, Dr. Alfred Sandrock, Biogen’s head of research and development, posted an open letter on the firm’s website in which he said the drug approval has been the focus of “extensive misinformation and misunderstanding.”

Sandrock, a key architect behind Aduhelm, the first new Alzheimer’s drug to win approval since 2003, said Biogen executives did nothing wrong by working closely with federal regulators.

“It is important to recognize that collaboration between industry and regulatory agencies is common, appropriate and beneficial,” he said, adding that Biogen welcomes the federal investigation.

He also said the agency was right to clear the drug under an “accelerated approval” pathway reserved for medicines that fill a serious unmet need. The agency uses that approach when regulators are uncertain about the clinical benefits of a drug and rely on another measure called a “surrogate biomarker” to determine whether the medicine might help patients.

In this case, the surrogate biomarker was how much Aduhelm reduced a sticky substance called amyloid that clumps into plaques in the brains of people with Alzheimer’s. Some doctors think it is the amyloid buildup that causes cognitive impairment, but other experts say that remains unproven.

Since 1992, Sandrock said, the FDA has granted 253 accelerated approvals for drugs to treat HIV and AIDS, sickle cell anemia, Duchenne muscular dystrophy, and, in particular, forms of cancer. In oncology, the surrogate measure may be tumor shrinkage, as that is likely to predict increased survival, he said.

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However, Sandrock did not to address a central criticism of the approval process: that the FDA previously did not consider the removal of amyloid plaques as “reasonably likely” to predict a clinical benefit, the standard for accelerated approval.

An independent advisory panel of experts to the FDA voted overwhelmingly in November that because of the conflicting results in the two large trials, there was insufficient evidence to recommend Aduhelm’s approval. After the FDA disregarded the panel’s recommendation last month, three members resigned in protest.

Brian Skorney, an analyst at the investment bank Baird, on Thursday rejected Biogen’s analogy that a reduction in plaque was a valid way to judge the likely effectiveness of an Alzheimer’s drug, much as a shrinking tumor was a valid way to judge the likely effectiveness of a cancer drug. In contrast to what doctors know about malignant tumors, Skorney said, physicians still don’t know whether plaque is a cause or effect of Alzheimer’s.

“I thought it was overly defensive,” Skorney, a critic of Aduhelm’s approval, said of Biogen’s presentation. “They feel embattled and feel it necessary to respond to all the critiques. I don’t think their arguments are entirely persuasive.”

Several prominent health systems, Cleveland Clinic, New York City-based Mount Sinai Health System, and Providence of Renton, Wash., have said they will not administer Aduhelm, citing the federal investigation, potential side effects such as brain swelling, and a lack of compelling clinical evidence that it works.

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The drug was commercially available for only a few weeks of the second quarter, and one of the biggest questions is how Medicare will cover it. The Centers for Medicare & Medicaid Services recently began a review of Medicare coverage, a process expected to take about nine months.

The vast majority of the roughly 6 million Americans with Alzheimer’s disease — 96 percent by one estimate — are over age 65 and would rely on Medicare as their primary insurer for coverage. Biogen estimates there are 1 million to 2 million people who have early symptoms and might benefit from the drug, which the FDA recently recommended that doctors start patients on only if they have mild cognitive impairment.

Nonetheless, many Medicare recipients would rely on secondary private insurance for other costs associated with Aduhelm. Several affiliates of Blue Cross and Blue Shield around the country have said they don’t plan to cover the medicine because they consider it “investigational” or “experimental” or because “a clinical benefit has not been established.”

About a third of the roughly 900 hospitals and clinics that Biogen expects will administer monthly intravenous infusions of Aduhelm have cleared it through internal review processes for adding it to their list of available medicines or said they won’t require a review, according to Alisha Alaimo, president of Biogen US.

“It’s still early days,” added Vounatsos.

Doctors involved in an internal review at Mass General Brigham, the largest health provider in Massachusetts, say the process won’t be completed until the fall.

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On the financial front, Biogen reported sales of $2.78 billion in the second quarter and earnings of $5.68 a share. The company expects revenue for the year to be between $10.65 billion and $10.85 billion, up from its previous guidance of between $10.45 billion and $10.75 billion. Since market close on June 7, Biogen’s stock is down nearly 18 percent, compared to a 3.3 percent increase in the S&P 500 Index.

Larry Edelman of the Globe staff contributed to this report.


Jonathan Saltzman can be reached at jonathan.saltzman@globe.com.