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USA: drug to control Alzheimer’s is approved by FDA panel

On Monday (10), a panel of independent consultants to the United States Food and Drug Administration (FDA) voted in favor of drugmaker Eli Lilly’s drug Donanemab, a monoclonal antibody designed to slow the progression of Alzheimer’s disease in the early stages. symptoms, agreeing that it is 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 remove a buildup of amyloid plaques in the brain, which is a hallmark of Alzheimer’s disease. These plaques are harmful deposits that can form in the brain as people age. Removing the plaque appears to slow brain changes related to Alzheimer’s disease.

Upon entering the brain, the medicine binds to amyloid plaques and its presence attracts the attention of the immune system, which facilitates the removal of the plaques.

The disease still has no cure, and there is no drug that prevents the development of Alzheimer’s, but Donanemab and Leqembi, another drug that is already on the market, manufactured by Eisai and Biogen, have been shown to slow the progression of the disease in its stages. initials.

The drugmaker behind the drug told the FDA’s Central and Peripheral Nervous System Drugs Advisory Committee that its data from a late-stage clinical trial showed “highly significant results” for patients taking donanemab, where the risk of disease progression was reduced. 37% lower compared to patients who received placebo for a year and a half.

In general, serious adverse events were “comparable” across all groups in the studies and infrequent, the company Lilly told the committee, occurring in just 2% of patients. Patients who received Donanemab had a slightly higher mortality rate – 2%, compared to 1.7% in the placebo group of the study.

While using the drug, three people died after developing ARIA, or microbleeds known as amyloid-related imaging abnormalities.

In trials of Leqembi, the drug that is already on the market, some patients presented ARIA, but the rate was lower than that observed in the Donanemab trial. Leqembi has also been linked to patient deaths.

Because most Donanemab patients who experienced problems with ARIA did so within the first six weeks of the study, Lilly told the committee that it added another MRI to the study to look for the problem before giving the patient a second infusion. The objective would be to detect patients with asymptomatic ARIA. If the problem was detected, providers would stop treatment so that it could be resolved and not become a more serious or symptomatic problem.

Analyzing these late-stage clinical trial results, the FDA committee voted on two specific questions and discussed three other key topics.

The committee voted a unanimous “yes” to questions about whether the data demonstrated that the drug is effective for treating Alzheimer’s disease and whether the drug’s benefits outweighed the risks in treating Alzheimer’s disease in the population enrolled in the clinical trials.

The FDA will take the committee’s recommendations into consideration when deciding whether to greenlight the drug.

Committee member Dr. Dean Follmann, assistant director of biostatistics at the National Institute of Allergy and Infectious Diseases, said he voted in favor because the evidence “was strong and consistent across all subgroups.”

In voting in favor, Sarah Dolan, acting consumer representative on the committee and consultant at the Critical Path Institute in Tucson, Arizona, said she was pleased with the survey results.

“There is a huge unmet medical need here that we hope can be addressed,” Dolan said.

Dr. Kathleen Poston, director of the Stanford Movement Disorders Center and a member of the committee, said she voted in favor because she believes the drug has benefits, “as long as the risks are monitored.”

The tests were carried out only with patients who had low and medium levels of tau, a protein that can become abnormal and contribute to the disease. The pharmaceutical company excluded those who did not have tau or had very low tau because the study would not follow the patients long enough to see if the drug made any difference. They also excluded people with high amounts of tau. The committee was concerned about extrapolating the results to a subset of patients with other tau levels; they wanted to see more data.

The majority of the committee was also uncomfortable with the FDA’s requirement that a tau PET test be needed to determine whether a patient was eligible to take the drug, since the test is not widely available and would make it difficult for people to access it. some people to the medicine.

The FDA also asked the committee to consider additional factors about ongoing research and factors related to potential administration of the drug.

The drug company Eli Lilly suggests that patients can stop taking the drug if they make enough progress. The committee suggested that research would have to show what the appropriate criteria would be for stopping or restarting the medication if symptoms returned.

The committee also noted that there was little diversity in the study, the majority of participants were white, and said future research will need to include more people of color to understand how the drug works in diverse populations. It also noted that if the drug is approved, doctors will have to educate their patients about the benefits and risks of taking that drug.

Initially, when the FDA said it would hold this meeting, the announcement came as a surprise, as many experts thought the drug would have been approved by the FDA last year.

Before the vote, Dr. Reisa Sperling, a professor of neurology at Harvard Medical School, told the committee that in the absence of treatments, the growing number of people with Alzheimer’s could become a serious threat to the country.

“We are doing such a good job of keeping people alive longer that we are creating a public health emergency if we don’t find a way to prevent this disease,” Sperling said.

Meanwhile, Eli Lilly said it is working on other treatments for Alzheimer’s.

“Recognizing the enormous burden this disease entails, we take very seriously the responsibility of bringing patients a well-characterized disease-modifying therapy,” Dr. David Hyman, chief medical officer of Eli Lilly and Company, told the committee.

The pharmaceutical company considers Donanemab as “an important but ultimately incremental step” in the treatment of 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 that drug.

“Patients deserve better and we continue to work on additional approaches to treat this disease,” Hyman said.

After the meeting, Eli Lilly said it was happy with the committee’s positive vote.

“We are pleased with the committee’s unanimous recognition of the positive risk and benefit profile of Donanemab,” said Mark Mintun, vice president of the drugmaker’s neuroscience research and development group in a press release. “We look forward to bringing this treatment option to patients.”

The Alzheimer’s Association asked the committee to recommend approval of Donanemab, and welcomed the committee’s unanimous vote in favor of the drug.

“A future with more approved treatments for Alzheimer’s disease is a tremendous step forward for the people eligible for these medicines,” said Dr. Joanne Pike, President and CEO of the Alzheimer’s Association. “Progress in treatment is happening.”

The national nonprofit organization USAgainstAlzheimer, which testified in favor of the drug during the public comment period at the meeting, said there is an urgency and a “huge unmet need” for Alzheimer’s treatments.

“If approved by the FDA, Donanemab will be the second drug on the market to help people in the early stages of Alzheimer’s disease have a better quality of life for a longer period, as well as offering patients and their doctors another option for their treatment plans,” said president and co-founder George Vradenburg. “We are grateful to the advisory committee for supporting this promising treatment.”

Source: CNN Brasil

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