Biogen Feeling the Heat

UPDATED: Biogen narrows its Aduhelm label after FDA left the floodgates open
Zachary Brennan
Senior Editor

While the FDA had controversially approved Biogen’s new Alzheimer’s drug Aduhelm with a wide label for anyone with the debilitating disease, Biogen announced Thursday morning that it’s narrowing the drug’s label as part of an attempt to calm at least some of its fiercest critics.

The unprecedented move means that the label now says that Aduhelm should be initiated in patients with mild cognitive impairment due to Alzheimer’s disease or mild Alzheimer’s dementia, which is reflective of the population in Biogen’s failed but resurrected clinical trials for the drug. The narrowed population may limit how much Medicare ends up spending on the drug, although if even a fraction of those with Alzheimer’s gain access to the drug, Biogen is likely to reap tens of billions of dollars per year from it.

“This is one of the most substantial labeling changes I can recall, and it is absent any new data,” Peter Bach, former drug pricing researcher and current CMO of Delfi Diagnostics, wrote on Twitter. “Parsimonious explanation: The FDA is a political agency that just happened to misread the politics.”

Bach added in a note to Endpoints News, “I was just thinking millions of Alzheimer’s patients had a drug for their disease yesterday who do not today, based on absolutely no new information. Even read most charitably this whole process from the calling [Biogen] back in to today is just entirely inept and awful for signaling regulatory consistency and reliability, never mind the bankrupting patients and Medicare with a toxic ineffective drug part of it.”

The updated label now reads (with changes in italics):

“ADUHELM is indicated for the treatment of Alzheimer’s disease. Treatment with ADUHELM should be initiated in patients with mild cognitive impairment or mild dementia stage of disease, the population in which treatment was initiated in clinical trials. There are no safety or effectiveness data on initiating treatment at earlier or later stages of the disease than were studied. This indication is approved under accelerated approval based on reduction in amyloid beta plaques observed in patients treated with ADUHELM. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s).”

John LaMattina, former Pfizer exec and current partner at PureTech Health, added, “Clearly the right thing to do and it should have been done in the original label. One has to wonder how the FDA screwed up on this. Leadership void? Pandemic fatigue?”

Rachel Sachs, a law professor at Washington University in St. Louis, also pointed out how the label change may not exactly limit those who would use Aduhelm, as it still says the drug is indicated for the treatment of Alzheimer’s disease. “One question to consider more closely is how and whether Aduhelm’s label has actually been narrowed. The new label says that treatment ‘should be’ focused on particular patients. But it still says treatment is ‘indicated for’ the broader Alzheimer’s population,” she said.


Al Sandrock
Biogen’s abrupt retreat is another odd turn in a series of bizarre moves from the biopharma giant, which is under intense congressional pressure on not just the accelerated approval but its dealings with the FDA prior to the sign-off and the eye-popping $56,000 per year price tag. While three of the adcomm members who voted almost unanimously to reject the drug have since resigned, FDA’s acting commissioner Janet Woodcock has defended the approval, while noting some “process problems,” including the fact that the committee never discussed the potential for an approval under the accelerated pathway.

Critics have also balked at the nine-year timeline for Biogen to run its confirmatory trial to prove that the drug actually shows clinical benefit. And in another unprecedented move last month, senior FDA leaders including CDER director Patrizia Cavazzoni, her new deputy Jacqueline Corrigan-Curay, and OND director Peter Stein met with Biogen to discuss that confirmatory study.

Al Sandrock, head of R&D at Biogen, said in a statement, “Based on our ongoing conversations with prescribing physicians, FDA and patient advocates, we submitted this label update with the goal to further clarify the patient population that was studied across the three ADUHELM clinical trials that supported approval. We are committed to continue to listen to the community’s needs as clinical practice adapts to this important, first-in-class treatment option.”