The future of Biogen’s potential blockbuster Alzheimer’s treatment was thrown back into doubt on Friday when an unusually combative scientific panel contradicted the positive conclusions of staff at the US drug regulator.
The panel voted that the company’s main trial data did not provide enough evidence that the drug, aducanumab, really worked.
Along with millions of Alzheimer’s sufferers and their loved ones, investors have been hanging on to the deliberations over aducanumab, which would be the first new treatment for the disease since 2003 if it is approved in the US.
Just two days ago, Biogen shares soared 44 per cent when the Food and Drug Administration’s own scientists said there was “substantial evidence” that aducanumab was effective.
At a meeting of the FDA’s advisory panel on Friday, however, many scientists derided the trial data as incomplete and inadequate, and some rounded on the regulator for pushing forward with the approval process.
Scott Emerson, emeritus professor of biostatistics at the University of Washington, accused Biogen of cherry-picking data to prove the drug works, likening it to “someone first firing a shotgun at a barn and then painting a target around the bullet holes”.
Caleb Alexander, professor at Johns Hopkins Bloomberg School of Public Health, said the FDA’s assessment appeared to be using the same language as Biogen had provided.
Joel Perlmutter, a professor of neurology at the Hope Center for Neurological Disorders, accused the FDA of trying to present the data to the panel as if “there was a bias in that one direction”.
Ten members of the 11-person panel voted that there was not enough evidence in the study to prove the drug was effective. One member voted “uncertain”.
Michel Vounatsos, Biogen’s chief executive, said it “appreciated the opportunity” to share data with the committee and thanked the patients who shared their experiences.
The FDA did not immediately respond to a request for comment.
The ultimate decision will be led by Billy Dunn, the acting director of the FDA’s office of neuroscience, who is not bound by the views of the advisory panel.
Speaking on Friday before the experts voted, he said he was “highly sensitive” to the “urgent need” for more drugs for Alzheimer’s, and that the data from the successful study were “strongly supportive” of approval.
In March 2019, Biogen halted both trials of aducanumab after an independent committee said it was not going to be effective. But it surprised investors in October last year when it claimed a further analysis showed the drug worked when taken at a higher dose.
Biogen is applying for approval based on one successful trial, with a subset of data from another trial that missed its goal. Advocates including Public Citizen and the National Center for Health Research urged the FDA to require Biogen to conduct a new trial.
Aaron Kesselheim, professor of medicine at Harvard Medical School, said there were “red flags” in the data, noting that the first study had not even finished. “It’s strange to rely on half or two-thirds of a study as your evidence of effectiveness for a drug,” he said.
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