Biogen Faces FDA Probe of Alzheimer’s Drug Approval; Stock Falls

(Bloomberg) -- The head of the U.S. Food and Drug Administration said she is seeking a federal investigation of the approval of the Biogen Inc. Alzheimer’s disease drug Aduhelm, a highly unusual step that will increase scrutiny of a heavily criticized clearance.

In a letter posted on Twitter, Janet Woodcock, the agency’s acting commissioner, said she was requesting an independent review of whether any interactions between Biogen and FDA staff were inconsistent with the agency’s policies and procedures. The inspector general for the Department of Health and Human Services would undertake the probe.

Biogen shares were down 3.5% at 3:34 p.m. Friday in New York.

Aduhelm was granted approval by the FDA last month over the objection of outside scientific advisers who had voted against the drug last November. The agency ordered Biogen to do an additional trial to confirm that the therapy benefits patients, and given nine years to produce data.

Biogen has priced Aduhelm at $56,000 annually.

In the letter, Woodcock said she had “tremendous confidence in the integrity” of the FDA staff, but said there were concerns over contacts between the agency and Biogen that may have occurred outside of the normal formal correspondence process.

“It is critical that the events at issue be reviewed by an independent body,” she wrote.

A Biogen spokeswoman said that the company would cooperate with any inquiry into the Aduhelm review process.

House Democrats are investigating the approval and pricing of Aduhelm, and senators have asked Finance Committee Chairman Ron Wyden to hold a hearing on the matter.

Democratic Senator Joe Manchin, considered a crucial vote in the party’s slim majority, blasted the approval and wrote President Joe Biden to request that someone other than Woodcock be chosen to be the agency’s permanent commissioner.

Frequent Meetings

Big trials of Aduhelm that were halted by Biogen in 2019 produced contradictory results as to whether it slows cognitive decline. So the agency instead approved the drug under a special pathway that requires less data called accelerated approval. This possibility wasn’t considered by the outside advisers who reviewed the drug’s data in November 2020.

Throughout 2019, after Biogen had said it had stopped its trials because the drug was unlikely to work, FDA officials held regular meetings with company executives, according to a person familiar with the matter.

Billy Dunn, the director of the FDA’s Office of Neuroscience, was central to the discussions. In the spring, Dunn met with Al Sandrock, who now serves as Biogen’s head of research and development, at a medical conference in Philadelphia, according to the person, and the regulator suggested Biogen not abandon the candidate.

FDA staffers later prepared a memo for a June 14, 2019, meeting with Biogen in which regulators and executives would discuss five different paths forward for Aduhelm, according to a person familiar with the matter. One of the pathways described in the memo included an accelerated approval, the person said.

The memo said that another alternative was to stop all clinical studies if the parties were to conclude that the evidence showed Aduhelm didn’t appear to work. FDA officials, however, said that option was off the table, according to the person, and proceeded to discuss alternatives.

Representatives for the FDA didn’t immediately respond to requests for comment. Dunn couldn’t immediately be reached for comment.

Some of the details of the meetings between company officials and the FDA were first reported by the news outlet Stat.

Accelerated Pathway

Five days later, in a meeting of Biogen’s board of directors, the accelerated approval pathway was once again discussed, said the person. The consensus among Biogen executives was that it was more likely that the experimental drug could gain approval through other means, such as pursuing a standard full clearance, or conducting another study.

Throughout the summer and fall, near-daily meetings between agency and company personnel, often held by phone, were described internally as a “working group” of sorts to sift through and analyze data, according to the person.

Biogen announced in October 2019 that it was reviving the program, but big trials sponsored by the company have produced conflicting results as to whether the drug can slow cognitive decline. One showed no effect and the other showed a very modest benefit.

Last November, a committee of FDA advisers voted nearly unanimously against the drug. But in June, Aduhelm received an accelerated approval on the basis of its ability to remove a bad protein called amyloid from the brain.

Biogen will still have to conduct another study to prove that the drug slows cognitive decline, but it can sell the drug in the meantime.

The agency hadn’t discussed publicly the possibility of an accelerated approval prior to its decision. The relationship between amyloid and cognitive decline is anything but clear-cut. Following the approval, three members of the advisory committee quit in protest.

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