Eli Lilly’s Alzheimer’s drug receives support from US advisory panel

Eli Lilly’s Alzheimer’s treatment donanemab is getting closer to US approval after an independent panel of scientific experts determined that the benefits outweighed the risk.

The US Food and Drug Administration convened a committee of independent experts to advise them on the effectiveness of donanemab as a treatment for dementia.

They cautioned that more data is needed to understand the effects of this drug in patients who have low or no tau levels, a protein associated with Alzheimer’s disease, since Eli Lilly did not include them in the clinical trial.

Costantino Iadecola is a Cornell University professor and a member of the committee. He said that if there are subgroups for which further analysis may be required, it should not delay making this drug accessible to the public.

Nilufer Ertekin Taner, a Mayo Foundation neurogeneticist, said that more data is needed for certain groups such as African Americans, Latin Americans, and people with Down syndrome.

The panel’s recommendation is not legally binding, but regulators usually follow the advice of such panels. The FDA requested the review in March delaying the approval for the treatment because it was concerned about the safety.

The vote was a positive for the largest drugmaker in the world, whose stock closed at 1.8% on Monday. Mark Mintun said that the US group was pleased with the unanimous recognition by the committee of the positive benefit-risk ratio of donanemab.

Eli Lilly’s drug belongs to a new treatment class that can slow down cognitive decline among patients with Alzheimer’s in the early stages by removing the amyloid plaques linked to this disease. Alzheimer’s affects 7mn Americans, and 50mn others worldwide. Until now, there have been few treatment options.

Analysts predicted for a long time that donanemab, which had shown a greater efficacy during clinical trials, would be superior to Leqembi. This was because donanemab required monthly infusions rather than twice a month. Leqembi was developed by Eisai in collaboration with Biogen. It uses a new mechanism to remove the amyloid plaque from the brain.

Three people have died as a result of brain swelling incidents in the trials of Eli Lilly’s drug.

Leqembi reduced cognitive decline in a phase three trial by 27% compared to the placebo group. Donanemab reduced cognitive decline by 35%, but with an increased incidence of rare side effects such as brain swelling. Eli Lilly developed its own cognitive test to gauge the trial’s effectiveness.

In a document released before the meeting on Monday, FDA staffers suggested that Eli Lilly’s drug could require a “black-box” warning to help patients understand its possible side effects. The FDA staffers also questioned if a tau level threshold should be used for determining which patients are offered treatment.

Leerink analysts cut their projections of donanemab sales for 2030 to $500mn last week, suggesting that it may not reach the “blockbuster status” of $1bn per year and will be well below analyst consensus estimates of $3.7bn.

People familiar with the situation say that Eli Lilly was taken by surprise when the FDA decided to request outside experts to review the treatment for Alzheimer’s.

Leqembi’s approval was also subjected to scrutiny by a committee of advisors. The controversy surrounding the accelerated approval for Aduhelm, the first Alzheimer’s drug Eisai and Biogen jointly developed, led to the resignation of three members from the FDA advisory committee and criticism by two congressional committees. Biogen gave up the rights to Aduhelm in January.