Eisai Co. and Biogen Inc.'s Alzheimer's drug Leqembi has received full approval from the Food and Drug Administration (FDA), making it the first disease-slowing medication to secure such a blessing. This development is expected to expand the availability of Leqembi to patients who are eagerly awaiting new and improved options to combat the progression of Alzheimer's disease. However, the implementation of this drug is likely to take longer than desired, as it requires specialized expertise and infrastructure to administer.
Peter Ljubenkov, a neurologist at the UCSF Weill Institute for Neurosciences, is excited about the approval and the opportunity to offer his patients Leqembi. However, he emphasizes that a significant amount of expertise and infrastructure are needed to properly administer the drug. Efforts have been underway for months to prepare the University of California hospital network for the arrival of Leqembi, but this is still a work in progress at memory centers across the country.
Despite Leqembi receiving conditional approval from the FDA in January, its usage was severely limited due to the Centers for Medicare & Medicaid Services (CMS) refusing to cover the drug's cost until it obtained full approval based on evidence of its ability to slow down the progression of the disease. With today's announcement, Leqembi has cleared that hurdle. However, there are still many pieces that need to fall into place before most patients can access it.
One of the hurdles is identifying the right patients. To be eligible for Leqembi, individuals must be in the early stages of Alzheimer's and have confirmation of the disease rather than another form of dementia. This requires objective evidence that their brains contain amyloid protein clumps, which Leqembi helps to remove. Detection methods include a special PET scan or a lumbar puncture to analyze cerebral spinal fluid. Unfortunately, amyloid PET scans, the less invasive method, are not covered by Medicare, but Leqembi's approval undermines the argument against coverage. It is crucial for CMS to agree to cover these scans promptly.
Another hurdle is the need for neurologists to have detailed discussions with patients about the potential risks of Leqembi. Patients taking blood thinners or those with two copies of the ApoE4 gene are at higher risk of experiencing more serious brain bleeding and swelling. This side effect has been underscored by the FDA, which added a black box warning to the drug's label. Additionally, patients will require an MRI to ensure the absence of brain bleeding before treatment begins, followed by several more scans during treatment to monitor for swelling or bleeds.
Lastly, there is the obstacle of Medicare's requirements for coverage. CMS will only cover Leqembi and similar drugs if real-world data on their efficacy and safety is collected, rather than relying on clinical trial results. Physicians will need to register patients in a registry that is yet to be determined. The complexity of this registry may slow down the process of providing the drug to patients in an equitable manner.
It is anticipated that the demand for the drug may also create challenges. With the availability of a treatment for mild Alzheimer's, individuals experiencing early symptoms may be more motivated to seek a diagnosis, potentially leading to an influx of patients at memory clinics. Additionally, there is concern among physicians that Leqembi may be prescribed too readily without a full understanding of its implications, similar to the overprescription of the previously approved Alzheimer's drug, Aduhelm.
Nevertheless, the FDA's decision marks an important milestone in the treatment of Alzheimer's. It sets us on the path towards developing a better system for caring for patients as future drugs emerge. While there may be initial challenges, let's hope that this is just the beginning of a new era in Alzheimer's treatment.