The U.S. Food and Drug Administration gave the go-ahead for Leqembi (lecanemab-irmb) to treat Alzheimer’s disease through the Accelerated Approval pathway. Leqembi is the second drug in a new class of Alzheimer’s drugs that are approved and work by changing the way the disease works. These medicines are an important step forward in the fight to treat Alzheimer’s disease effectively.
Billy Dunn, M.D., director of the Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research, said, “Alzheimer’s disease destroys the lives of those who have it and the lives of those who love them.” “This is the newest way to treat Alzheimer’s. Instead of just treating the symptoms, it targets and affects the disease process itself.”
Alzheimer’s disease is an irreversible, progressive brain disorder that affects more than 6.5 million Americans. It slowly destroys memory, thinking skills, and, eventually, the ability to do simple things. Even though we don’t fully understand what causes Alzheimer’s, we do know that it is marked by changes in the brain, such as amyloid beta plaques and neurofibrillary, or tau, tangles, which cause neurons and the connections between them to die. These changes affect how well a person can think and remember.
Leqembi was approved through the Accelerated Approval pathway. Under this pathway, the FDA can approve drugs for serious conditions where there is an unmet medical need and a drug has been shown to have an effect on a surrogate endpoint that is likely to predict a clinical benefit to patients. The results of a Phase 3 randomised, controlled clinical trial to confirm the drug’s clinical benefit were just released, and the agency expects to get the data soon.
In a double-blind, placebo-controlled, parallel-group, dose-finding study with 856 Alzheimer’s disease patients, researchers looked at how well Leqembi worked. When the presence of amyloid beta pathology was confirmed, treatment was given to people who had mild cognitive impairment or mild dementia. Patients who got the treatment had a significant dose- and time-dependent reduction in amyloid beta plaque. Patients who got the approved dose of lecanemab, 10 milligrammes per kilogramme every two weeks, had a statistically significant reduction in brain amyloid plaque from baseline to Week 79, compared to the placebo arm, which had no reduction in amyloid beta plaque.
These results back up the faster approval of Leqembi, which is based on the fact that amyloid beta plaque, a sign of Alzheimer’s disease, has been seen to decrease. The amount of amyloid beta plaque in the brain was measured with positron emission tomography (PET) imaging. This was done to compare the levels of amyloid beta plaque in a group of brain regions that are likely to be affected by Alzheimer’s disease pathology to a group of brain regions that are likely to be unaffected.
In the information about how to give Leqembi, there is a warning about amyloid-related imaging abnormalities (ARIA), which can happen with antibodies in this class. Most people with ARIA don’t have any symptoms, but sometimes serious and life-threatening things can happen. Most people with ARIA have temporary swelling in parts of the brain, which usually goes away on its own. Some people may also have small spots of bleeding in or on the surface of the brain, headaches, confusion, dizziness, changes in vision, nausea, and seizures. Leqembi also comes with a warning that there is a chance of reactions caused by the infusion, such as flu-like symptoms, nausea, vomiting, and changes in blood pressure. Most people who took Leqembi had reactions to the way it was given, headaches, and ARIA.
According to the information on how to prescribe Leqembi, it is used to treat Alzheimer’s disease. The label says that Leqembi treatment should start with people who have mild cognitive impairment or mild dementia. This is the group of people who were studied in clinical trials. The label also says that there are no safety or effectiveness data about starting treatment earlier or later than what was studied.