Eli Lilly to apply for accelerated approval of unproven Alzheimer’s drug

Eli Lilly on Thursday said it plans to submit its Alzheimer’s drug to the Food and Drug Administration (FDA) for accelerated approval later this year, a move that takes advantage of the agency’s controversial approval of a similar drug from Biogen. 

The company in a statement said it intends to submit its drug called donanemab to the FDA by the end of the year, but it could happen much sooner.

Lilly said the agency gave donanemab a “breakthrough therapy” designation, which is meant to expedite the development and review of drugs that may demonstrate substantial improvement over already established therapies. 

The announcement from Lilly comes after the FDA overruled its own outside advisory panel to approve Biogen’s drug Aduhelm, saying it was “reasonably likely” the treatment would slow cognitive decline in patients.

FDA’s decision surprised many inside and outside the agency, and led some members of the panel to resign in protest. The agency has been under pressure to approve new Alzheimer’s drugs, since it had been almost 20 years since the last one arrived on the market.

Aduhelm was granted accelerated approval based on a “surrogate endpoint.” Instead of basing the decision on whether the drug slows cognitive decline, FDA instead granted approval based on the reduction of amyloid plaque in the brain.

The data from Biogen’s drug was confusing, at best, and many experts disagree that reducing plaque can actually help slow cognitive decline. But the FDA gave Biogen nine years for a confirmatory trial to prove how it can benefit patients. 

There are approximately 6 million Americans who suffer from Alzheimer’s, meaning Aduhelm is poised to be a windfall for Biogen, which priced its drug at $56,000 a year per patient. 

Lilly’s drug is also an amyloid-reducer, and the company said it will file based on the results of a mid-stage clinical trial involving 272 patients. The company is conducting a larger phase 3 trial, but Thursday’s announcement indicates the company is not going to wait for the results. 

FDA’s approval of Aduhelm essentially set the bar for evidence much lower for experimental Alzheimer’s drugs, and Lilly is poised to take advantage of the opening.

During an investor conference last month, Eli Lilly’s CFO Anat Ashkenazi said he expected to need a higher bar for approval than just a small, phase 2 trial. 

“The history … for this division of the FDA is that it requires two controlled studies for an approval, and we believe that’s still the case and should be the case, ” Ashkenazi said. “We believe that the path for approval is … the two well-controlled studies but if anything changes in two weeks [with Aduhelm] that we learn about then we’ll continue these conversations with the FDA.”