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On Monday, a panel of independent advisers to the U.S. Food and Drug Administration voted to approve Eli Lilly’s drug donanemab, a monoclonal antibody designed to slow the progression of early symptoms of Alzheimer’s disease, recognizing that it was safe and effective.
According to the Alzheimer’s Association, one in three older Americans dies from Alzheimer’s disease or another form of dementia, killing more people than prostate and breast cancer combined.
Donanemab works by helping the body clear the buildup of amyloid plaque in the brain that is characteristic of Alzheimer’s disease. These plaques are harmful deposits that can form in the brain as we age. Removing plaque appears to slow the brain changes that seem to occur with Alzheimer’s disease.
When the drug enters the brain, it binds to amyloid plaques and its presence attracts the attention of the immune system, making it easier to clear the plaque.
There is no cure and there is still no drug to prevent the development of Alzheimer’s disease, but donanemab and Leqembi, another drug already on the market made by Eisai and Biogen, slow the progression of the disease in its early stages. .
Lilly told the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee that its late-stage clinical research data showed “highly significant results” for patients taking donanemab, with a risk of progression of the disease 37% lower over a year and a half. compared to patients who received the placebo.
Serious adverse events were “generally comparable” between all groups participating in the trials and were uncommon, Lilly told the committee, occurring in only 2% of patients. Patients on donanemab had a slightly higher mortality rate – 2%, compared to 1.7% in the placebo arm of the trial.
Three people died while taking the drug after developing ARIAs, or microbleeds known as amyloid-related imaging abnormalities.
In trials of Leqembi, the drug already on the market, some patients suffered from ARIA, but at a lower rate than observed in the donanemab trial. Leqembi has also been linked to patient deaths.
Since most donanemab patients who experienced problems with ARIA did so within the first six weeks of the trial, Lilly told the committee that it added another MRI to the trial to look for the problem before giving the patient a second infusion. The objective would be to detect patients with asymptomatic ARIA. If detected, providers would stop treatment so that the problem can resolve itself and not become more serious or symptomatic.
In reviewing these late-stage clinical trial results, the FDA committee voted on two specific issues and discussed three other main topics.
The committee voted “yes” unanimously to questions about whether the data showed the drug is effective in treating Alzheimer’s disease and whether the benefits of the drug outweighed the risks in treating Alzheimer’s disease. Alzheimer’s disease in the population enrolled in clinical trials. .
The FDA will consider the committee’s advice when deciding whether to authorize the drug.
Committee member Dr. Dean Follmann, deputy director of biostatistics at the National Institute of Allergy and Infectious Diseases, said he voted yes because the evidence “was strong and consistent across all subgroups.” “.
Voting yes was Sarah Dolan, acting consumer representative on the committee and consultant to the Critical Path Institute in Tucson, Arizona., said she was satisfied with the results of the research.
“There is a huge unmet medical need here that we hope we can meet,” Dolan said.
Committee member Dr. Kathleen Poston, director of the Stanford Movement Disorders Center, said she voted yes, believing the drug has a benefit, “as long as the risks are monitored.”
The trials included only patients with low to medium levels of tau, a protein that can become abnormal and contribute to disease. Lilly excluded patients who had no or very little tau because the study would not follow patients long enough to see if the drug made a difference. They also excluded people with high amounts of tau. The committee had some concerns about extrapolating the results to a subset of patients with other tau levels; they wanted to see more data.
The majority of committee members also were not comfortable with the FDA requiring a Tau PET test to determine whether a patient was eligible to take the drug. since the test is not widely available and it would be difficult for some people to obtain the medication.
The FDA also asked the committee to consider additional factors regarding ongoing research and factors related to possible administration of the drug.
Lilly suggests that patients might be able to stop the drug if they show enough progress. The committee suggested that research should show what would be the appropriate criteria for stopping or restarting treatment if symptoms returned. The committee also noted that there was little diversity in the trial—most participants were white—and said future research will need to include more people of color to understand how the drug works in diverse populations. The committee also noted that if the drug is approved, doctors will need to educate their patients about the benefits versus risks of taking the drug.
Initially, when the FDA announced that it was going to hold this meeting, it was a surprise announcement since many experts believed the drug would have been approved by the FDA last year.
Before the votes, Dr. Reisa Sperling, a professor of neurology at Harvard Medical School, told the committee that without treatment, the growing number of people with Alzheimer’s disease could become a serious threat to the country.
“We’re doing such a good job of keeping people alive longer that we’re creating a public health emergency if we don’t find a way to stave off this disease,” Sperling said.
In the meantime, Lilly said it is working on other treatments for Alzheimer’s disease.
“Recognizing the enormous burden of this disease, we take very seriously the responsibility to provide patients with a well-characterized disease-modifying treatment,” Dr. David Hyman, chief medical officer of Eli Lilly and Company, told the committee. .
Lilly considers donanemab “an important, but ultimately progressive step” in treating Alzheimer’s disease. Ultimately, Hyman said Lilly would like to develop a drug that would treat the disease before someone even starts showing symptoms and is working on such a drug.
“Patients deserve more and we continue to work on additional approaches to combat this disease,” Hyman said.
After the meeting, Lilly said she was pleased with the committee’s positive vote.
“We are pleased with the committee’s unanimous recognition of donanemab’s positive benefit-risk profile,” said Mark Mintun, Eli Lilly group vice president, neuroscience research and development, in a press release. “We look forward to bringing this treatment option to patients.” »
The Alzheimer’s Association urged the committee to recommend approval of donanemab and welcomed the committee’s unanimous vote in favor of the drug.
“A future with more approved Alzheimer’s treatments is significant progress for those eligible for these medications,” said Dr. Joanne Pike, president and CEO of the Alzheimer’s Association. “Progress is being made in treatment. »
USAgainstAlzheimer’s, a national nonprofit organization, which testified in favor of the drug during the public comment period at the meeting, said there was an urgency and a “massive unmet need” for treatments against Alzheimer’s disease.
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“If approved by the FDA, donanemab would be the second drug on the market to help people in the early stages of Alzheimer’s disease experience a better quality of life for a longer period of time, while also providing patients with and their doctors another option for their treatment. plans,” said president and co-founder George Vradenburg. “We are grateful to the advisory committee for their support of this promising treatment. »
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