Today the US health officials approved a medicine meant to slow the progression of Alzheimer’s disease. The FDA accelerated approval of the new medicine known scientifically as lecanemab has drawn scrutiny given its potential wide use and its high cost of $26,500 per year for an average patient. “Leqembi is the second of a new category of medications approved for Alzheimer’s disease that target the fundamental pathophysiology of the disease” states the Press Release. In 2021, the FDA similarly cleared Aduhelm, developed through the same partnership between the Japan-based pharmaceutical company Eisai and US Biogen.
Fridays’ announcement by the FDA comes three months after a large trial findings that treatment with Leqembi slowed patients’ physical and cognitive decline over 18 months. Although the results were the first clear-cut success for a drug of Leqembi’s type in late-stage clinical testing, the accelerated approval requires companies to conduct another clinical trial before full approval can be considered. Still there are many unanswered questions about the drug with regard to the magnitude of its benefit, especially in light of its potential safety risks.
Importantly for the FDA’s decision-making, Leqembi worked as it was designed to — making it the first therapy approved in the U.S. for the treatment of Alzheimer’s itself by targeting the underlying disease process rather than symptoms of the disease.
Alzheimer is an irreversible, progressive brain disorder affecting more than 6.5 million Americans that gradually destroys memory and cognitive skills eventually affecting the ability to carry out simple tasks.
Researchers evaluated the efficacy of Leqembi in a double-blind, placebo-controlled, parallel-group, dose-finding study of 856 patients with Alzheimer’s disease, as specifies in the prescribing information. The labeling states that treatment with Leqembi should be initiated in patients with mild cognitive impairment or mild dementia stage of disease, the population in which treatment was studied in clinical trials.
The approval of the drug, lecanemab, to be marketed as Leqembi, is likely to generate considerable interest from patients and physicians, per news reporting. In its decision, the FDA appeared to acknowledge the criticism brought on by its approval of Aduhelm in 2021 after both a committee of independent advisers and a council of senior officials at FDA pointed out that the data failed to conclusively show that Aduhelm slowed cognitive decline.
With Leqembi, the F.D.A. included narrower and more cautionary language on the drug label than it initially had with Aduhelm. The Leqembi label says the drug should be used only for patients in early and mild stages of Alzheimer’s disease, matching the status of patients in the clinical trials of the drug. Data shows that about 1.5 million of the six million people with Alzheimer’s in the United States are estimated to be in the beginning phases of the disease, with diagnoses of either mild cognitive impairment or early-stage Alzheimer’s. The number of patients to be treated with Leqembi will depend significantly on whether Medicare covers the drug.
Leqembi, the brand name, is based on “qembi” in Japanese, which “roughly translates into beautiful, healthy, elegant”. Alzheimer’s is the most common form of dementia, yet much remains unknown about this progressive neurological disorder.
Source: FDA News Release