Leqembi and Down Syndrome
The NTG has accumulated a selective library of publications and news regarding Leqembi (lecanemab) and Down syndrome.
On January 6, 2023 the U.S. Food & Drug Administration (FDA) granted accelerated approval for lecanemab, an anti-amyloid drug designed to treat Alzheimer’s disease that was developed by the pharmaceutical firms Eisai and Biogen. Lecanemab, branded as Leqembi, was given full traditional approval on July 6, 2023 by the FDA to treat adults in the mild stage of Alzheimer’s dementia who also have “confirmed presence of amyloid beta pathology” in the brain. The presence of these amyloid beta plaques in the brain is one of the hallmarks of Alzheimer’s disease. An updated commentary on the FDA decision and subsequent release by CMS to have Medicare coverage for the drug is provided by Lumind IDSC. The NTG and Lumind IDSC are working cooperatively to ensure that state drug formularies do not include exclusions for access by adults with intellectual disability, including Down syndrome. A complementary effort is underway to include provisions for noting DS-AD and specialized DS-appropriate instruments on the CMS registry for Leqembi.
Relevant Publications
A Step Forward in the Fight Against Dementia -- Are We There Yet?
Journal article
This viewpoint reports on the results of the Clarity AD trial, a phase 3 randomized clinical trial of lecanemab for patients with early Alzheimer disease, in which lecanemab’s clinical efficacy was demonstrated using well-established outcome measures. Authors discuss the implications of the findings and the reported risks. From a clinician's perspective, they comment that persons diagnosed with Alzheimer's disease should have access to this drug, although no mention is made of its safety or applicability to adults with intellectual disability. The authors note the work that still remains to be undertaken, lecanemab's success represents a major milestone for the field, and what they consider as a moment of great hope for patients and families living with Alzheimer's disease.
Source: Wolk DA, Rabinovici GD, Dickerson BC. A Step Forward in the Fight Against Dementia-Are We There Yet? JAMA Neurol. 2023 Mar 13. doi: 10.1001/jamaneurol.2023.0123. Epub ahead of print. PMID: 36912845.
Adapting Eligibility Criteria for Prescribing FDA Approved Anti-Amyloid Immunotherapeutics for Adults with Down Syndrome with Early-Stage Alzheimer’s Dementia (Statement)
Statement
An Advisory and Consensus Statement of the Working Group on Criteria for Access to Alzheimer’s Therapeutics for Adults with Down Syndrome; experts were convened to determine prescribing criteria equivalences that would be inclusionary of adults with Down syndrome. This advisory and consensus statement is the result of the experts’ deliberations and recommendations for addressing this inequity to treatment access and includes alternative inclusionary language and modified criteria, as well as providing a roadmap for prescribers when determining eligibility for adults with Down syndrome.
Down syndrome in a new era for Alzheimer Disease
Journal article
This Viewpoint explains the genetic association between Alzheimer disease and Down syndrome and the negative impact of excluding people with Down syndrome from clinical trials on treatment for Alzheimer disease.
Source: Rafii MS, Fortea J. Down Syndrome in a New Era for Alzheimer Disease. JAMA. 2023 Dec 12;330(22):2157-2158. doi: 10.1001/jama.2023.22924. PMID: 37991807.
F.D.A Widens Access to Leqembi Drug for Alzheimer's
Newspaper article
The Food and Drug Administration on July 6, 2023 gave full approval to the Alzheimer’s drug Leqembi, and Medicare said it would cover much of its high cost, laying the foundation for widespread use of a medication that can modestly slow cognitive decline in the early stages of the disease but also carries significant safety risks. Leqembi will be available for people with mild dementia or a pre-Alzheimer’s condition called mild cognitive impairment. The F.D.A. label instructs doctors not to treat patients without testing to confirm they have an accumulation of the protein amyloid, a hallmark of Alzheimer’s that Leqembi attacks. The F.D.A. decision granting full approval to Leqembi means that Medicare will cover it for eligible patients. Some patients will be unable to afford the 20 percent Medicare does not cover, possibly about $6,600 a year. Including costs of medical visits and required regular brain scans, some of which will receive Medicare reimbursement, the treatment could run to about $90,000 a year. Doctors prescribing Leqembi will be required to submit medical information about each patient before and while they are being treated with the drug. The information will be kept in patient registries and evaluated to learn more about Leqembi’s benefits or harms.
F.D.A. Approves Lecanemab as New Treatment for Early Alzheimer’s
Newspaper article
Food and Drug Administration on January 7, 2023, approved another Alzheimer’s drug that may modestly slow the pace of cognitive decline early in the disease, but also carries risks of swelling and bleeding in the brain.
Lecanemab For Treating Alzheimer’s Disease: New Clinical Trial Results from Patients with Early-Stage Disease
Statement
LuMind IDSC has noted that there remain substantial barriers to Leqembi being deemed safe and accessible for people with Down syndrome, as no one with Down syndrome was included in the Leqembi clinical trials to date. LuMind IDSC recommends that a safety study specifically geared to people with Down syndrome should take place prior to widespread use in the Down community.