Alzheimer's Drug Leqembi Secures FDA Approval, Paving the Way for Medicare Coverage

The FDA gave provisional clearance to Eisai, a Japanese pharmaceutical firm. This decision was taken after a careful review of early data, which showed that Leqembi, their novel medicine, effectively cleared amyloid plaques linked with Alzheimer's disease.

The United States Food and Drug Administration (FDA) officially announced on Thursday, revealing their approval of the groundbreaking Alzheimer's medication known as Leqembi. This decision came after a rigorous confirmatory trial was conducted, successfully confirming the drug's significant clinical benefit. Consequently, it is imperative to incorporate the costly treatment into Medicare, the federal health insurance program designed for individuals aged 65 and above and younger individuals with disabilities.

Leqembi has become the inaugural amyloid beta-directed antibody to transition from an accelerated approval, granted in June, to a conventional approval to treat Alzheimer's disease. The drug's mechanism of action involves the reduction of amyloid plaques, which are known to form in the brain and are considered a key pathophysiological characteristic of the disease.

Leqembi received approval in January through the Accelerated Approval pathway, a regulatory pathway designated for medications addressing severe medical conditions that lack adequate treatment options. Based on an official statement from the FDA, the decision was made after careful consideration of clinical data that provided compelling evidence suggesting a high probability of predicting a tangible clinical benefit for patients. In accordance with subsequent regulatory guidelines, the FDA has mandated the execution of a confirmatory study. The trial's findings demonstrated the efficacy of Leqembi in modestly reducing the progression of Alzheimer's disease.

According to the FDA, Leqembi may cause several common side effects, including headaches, infusion-related reactions, and amyloid-related imaging abnormalities (ARIA). ARIA is a known side effect associated with antibodies that target amyloid plaques. ARIA typically manifests as temporary brain swelling, which can be observed through imaging studies. This swelling tends to subside naturally and may be accompanied by small instances of bleeding within or on the brain's surface. While ARIA typically does not present with noticeable symptoms, it is possible for symptoms to manifest, such as headaches, confusion, dizziness, changes in vision, and feelings of nausea. In rare cases, ARIA may manifest with a potentially life-threatening condition called brain edema. This condition can lead to seizures and other severe neurological symptoms.

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The FDA confirmed the results through a thorough analysis of data from a comprehensive study involving 1,800 patients. This study revealed that the drug effectively reduced the rate of memory and cognitive decline by approximately five months in individuals who received the treatment compared to those who were administered a placebo.

Alzheimer's disease is a degenerative condition of the brain that cannot be reversed and worsens over time. It impacts a significant number of individuals, with over 6.5 million Americans being affected by this disease. This disease gradually deteriorates memory and cognitive abilities, eventually impairing the individual's capacity to perform basic tasks. While the exact causes of Alzheimer's disease are not completely comprehended, it is distinguished by alterations in the brain. These changes involve the development of amyloid beta plaques and neurofibrillary tangles, also known as tau tangles. Ultimately, these changes lead to the deterioration of neurons and their connections.

In January, the FDA granted Leqembi accelerated approval. This type of approval is given to drugs that address serious conditions with unmet medical needs. It allows for earlier approval, even if further studies are still being conducted to confirm the expected clinical benefit. During a trial in its advanced stages, the treatment demonstrated a significant 27% reduction in the rate of decline in cognition and function compared to a placebo over 18 months. 

In early June, a group of FDA advisers unanimously supported using Leqembi. They based their decision on clinical studies that confirmed its effectiveness. However, during the public hearing, some experts expressed concerns about safety issues observed in the clinical trials, such as the possibility of brain swelling or bleeding. According to the FDA, the drug's label will include a boxed warning regarding amyloid-related imaging abnormalities. While these abnormalities typically do not present any symptoms, there have been rare instances where they have led to dangerous swelling in the brain, posing a potential threat to life.

In June, the Centers for Medicare and Medicaid Services announced that Medicare would provide coverage for emerging Alzheimer's treatments, including Leqembi. However, this coverage is contingent upon the drugs receiving formal FDA approval and the involvement of the beneficiary's doctor and clinical team in gathering real-world evidence on the drugs' effectiveness. According to a research note from BMO Capital Markets analysts in June, the recent announcement was considered a significant success for both Biogen and Eli Lilly. Eli Lilly is currently working on its treatment called donanemab in the same category. According to the report, analysts have estimated that Leqembi is expected to reach peak sales of $14 billion, while donanemab is projected to achieve peak sales of $17 billion. 

Policy experts have indicated that while Medicare does offer coverage, it may still be financially burdensome for numerous patients to afford Leqembi. Based on the health policy nonprofit KFF findings, Medicare patients who are prescribed this medication will be responsible for paying more than $5,000 annually from their personal funds. This is because, under traditional Medicare, there is a 20% coinsurance requirement for this particular drug, with a list price of $26,500. It is important to mention that the average income of individuals who benefit from Medicare is around $30,000. People with supplemental insurance, like Medigap, may have lower out-of-pocket expenses.

The inclusion of the new treatment in Medicare coverage has the potential to increase the overall expenditure in Medicare Part B greatly. This particular part of Medicare focuses on covering drugs, such as the infusion Leqembi, that are typically administered by healthcare professionals rather than being self-administered by patients. According to a recent analysis by KFF, if 10% of older adults with Alzheimer's disease were to take Leqembi, the annual Medicare spending on this drug would amount to approximately $17.8 billion. This figure represents nearly half of the total spending on the over 600 Part B-covered drugs in 2021, which amounts to $40 billion.

According to KFF, the increased spending could potentially result in higher Part B premiums. According to regulations, Part B premiums must be set at 25% of the estimated total expenses of the program. The premiums for Part B have increased by 15% for 2022. This increase is partly due to the uncertainty surrounding Medicare's potential spending on Aduhelm, a new treatment for Alzheimer's disease that received accelerated approval from the FDA in 2021. 2023 there has been a 3% decrease in Part B premiums. This reduction can be attributed, at least in part, to a decrease in the price of Aduhelm and Medicare's decision to restrict coverage for this particular drug.

Including a patient registry as a prerequisite for Medicare coverage of the latest Alzheimer's treatments has faced criticism from certain patient advocates and other organizations. They argue that it creates an unnecessary obstacle to accessing care. In a statement released in June, the Alzheimer's Association expressed their belief that while registries can be valuable in collecting real-world evidence to enhance patient care, they should not be obligatory for the coverage of FDA-approved treatments.

In late June, CMS announced that the information gathered would be used to assess the suitability of treatment for Medicare beneficiaries. According to the agency, healthcare providers administering the drug to patients must provide data regarding the outcomes of cognition and overall function tests. Additionally, they must report any instances of brain swelling, hemorrhage, other adverse events, and other relevant details.

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                      Boston neurologist discusses new Alzheimer's drug Leqembi