FDA approves Alzheimer’s drug Leqembi.

FDA approves Alzheimer's drug Leqembi.

FDA Grants Full Approval to Alzheimer’s Drug Leqembi

Alzheimer’s Disease

The Alzheimer’s drug Leqembi has received full approval from the U.S. Food and Drug Administration (FDA), a significant milestone that paves the way for insurance coverage of the expensive medication. The approval means that Medicaid and Medicare will cover the drug, as long as patients are enrolled in a registry to track their progress. The drug’s high cost of $26,500 per year has been a major barrier for many individuals with early-stage Alzheimer’s. Now, with insurance coverage available, more people can access this breakthrough treatment.

The Holy Grail of Alzheimer’s Treatment

The approval of Leqembi is a remarkable breakthrough in Alzheimer’s treatment because it is the first disease-modifying drug for the disease. For decades, researchers have been searching for a way to eliminate amyloid protein from the brain, as amyloid buildup is a key signal of Alzheimer’s. Leqembi is the first successful drug that has been shown to delay the progression of the disease by removing amyloid. During an 18-month trial, Leqembi demonstrated a five-month delay in Alzheimer’s progression, which is a significant improvement.

A Breakthrough with Caution

While the approval of Leqembi is a major step forward, it comes with a caveat. The FDA has added a black-box warning to the drug’s labeling, cautioning that it can trigger serious and life-threatening events, including brain bleeds. In rare cases, these events have proven fatal. Therefore, Leqembi will only be available to individuals in the earliest stages of Alzheimer’s, those with mild dementia or mild cognitive impairment. Physicians must also ensure that patients have already undergone testing to confirm elevated levels of amyloid protein in their brains before prescribing Leqembi.

A Promising Alternative to Aduhelm

Leqembi is only the second Alzheimer’s drug to receive FDA approval in the past two decades, with the controversial approval of Aduhelm in 2021. Unlike Aduhelm, which had incomplete data and lacked evidence of cognitive decline reduction, Leqembi showed statistically significant slowing in both cognitive and functional decline. In addition, it effectively reduced brain amyloid levels and showed downstream beneficial effects on other markers of neurodegeneration. Dr. Sarah Kremen, from Cedars-Sinai in Los Angeles, emphasized these positive outcomes when Leqembi received accelerated approval back in January.

Understanding the Risks

Although Leqembi has shown promise in clinical trials, two deaths from brain bleeds among participants have raised concerns. One case involved a 65-year-old woman who died from a massive brain bleed linked to Leqembi. Another case involved an 80-year-old man who experienced a possible interaction between Leqembi and a blood thinner he was taking. Autopsies revealed that both individuals had widespread cerebral amyloid angiopathy, a condition where amyloid deposits replace blood vessel walls. Stripping amyloid away can weaken blood vessels, making them susceptible to bleeds when exposed to blood thinners or clot busters. However, other factors may have contributed to these fatal outcomes.

Weighing the Benefits and Risks

Despite the risks, a beneficial Alzheimer’s drug is much needed, considering the millions affected by the disease. After Medicare limited coverage of Aduhelm, citing risks and unclear benefits, the drug’s availability was greatly diminished. Leqembi, like Aduhelm, targets amyloid proteins to reduce their clumping in the brains of individuals with Alzheimer’s. Although more research is needed to establish the long-term effects and efficacy of these drugs, the approval of Leqembi represents a significant step forward in the treatment of Alzheimer’s disease.

It’s important to note that future drugs with improved effectiveness will likely be developed, building upon the progress made with Leqembi. While there are still gaps in our understanding of Alzheimer’s and the best approaches for treatment, the approval of Leqembi offers hope for a longer period of independent living for patients.