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FDA issues approval for Kisunla, new Alzheimer’s drug; Down syndrome community optimistic

On Tuesday, July 2, the U.S. Food and Drug Administration (FDA) announced the approval of Kisunla (donanemab), an anti-amyloid drug developed by Eli Lilly and Company (Lilly) that has been shown in clinical trials to slow the progression of Alzheimer’s disease in the general population.

Kisunla joins previously approved Leqembi (Eisai) and Aduhelm (Biogen) as the first generation of Alzheimer’s therapies designed to remove brain plaque and subsequently slow cognitive and functional decline in people with early symptomatic Alzheimer’s disease.

Clinical trial data released by Lilly in May 2023 outlined the notable efficacy of Kisunla, with analysis showing trial participants retained their ability to perform the meaningful activities of daily living such as managing finances, driving, engaging in hobbies, and conversing about current events for a longer period of time compared to participants receiving placebo.

For the thousands of people diagnosed with Down syndrome-associated Alzheimer’s disease (DS-AD) and their families, today’s approval is a reason to hope and celebrate. It represents the third approval of a disease modifying anti-amyloid drug in three years and offers patients more options when and if they choose to take an Alzheimer’s therapy.  

Hampus Hillerstrom, CEO of LuMind IDSC, said the Down syndrome (DS) community is encouraged by the approval of Kisunla, because in a time when up to 90% of people with Down syndrome could receive an Alzheimer’s diagnosis in their lifetime, “every new treatment option that becomes available for Alzheimer’s disease is critically important for the Down syndrome community.”

He said the community’s enthusiasm for progress in Alzheimer’s treatment is met with an equal measure of frustration because of the remaining barriers currently impeding people with Down syndrome from accessing the new Alzheimer’s drugs due to the lack of safety data in adults with Down syndrome with these new therapies.

“Unfortunately, no one with Down syndrome was included in the clinical trials of Kisunla, nor in any of the previous trials in this drug class,” Hillerstrom explained. “So, we still don’t know if Kisunla or any other anti-amyloid drugs are safe for people with Down syndrome.” However, barriers to treatment are changing for the DS population. For example, the Chief Medical Officer of Eli Lilly, David Hyman, MD, announced at a recent FDA meeting that a clinical trial is being planned for the Down syndrome community. Also, the U.S. Centers for Medicare and Medicaid (CMS) confirmed that the DS population is covered by Medicare if prescribed Leqembi, and a workgroup led by LuMind IDSC and the National Task Group on Alzheimer’s and Dementia (The NTG) has shared its DS-AD adaptation recommendation to the CMS registry for anti-amyloid drugs.

Public calls for policy change are happening in parallel with the development of procedure adaptations to support clinical trials in the Down syndrome population.

What’s next for the Down syndrome community and the new Alzheimer’s treatments?

Safety Trials. We don’t know if Kisunla or any of the previously approved anti-amyloid drugs are safe for people with Down syndrome. 

A LuMind IDSC analysis of publicly available data shows that no one with Down syndrome was included in any of the clinical trials to date, so the safety profile of Kisunla in the Down syndrome population remains unknown.

The Phase 3 results of the Kisunla trial demonstrated there is more work to be done to understand the implication of amyloid plaque presence in the brain and the incidence of amyloid-related imaging abnormalities, ARIA. The most common ARIA is a temporary swelling in an area or areas of the brain (ARIA-E) or a small brain bleed “microhemorrhages” or a more severe brain bleed, “superficial siderosis” (ARIA-H).  Read more about the concerns around ARIA here.

LuMind IDSC and our partners are strongly advocating and working to prepare the pharmaceutical industry for the trial to take place as soon as possible.  You can read more about those efforts on our web site.

CLINICAL & COMMUNITY PREPARATIONS: In addition to these considerations, the drug companies will aim to ensure that the trial is as safe as possible for our population, that the clinical trial sites are ready and trained to work with people with Down syndrome, and that outreach videos and materials are adapted for the Down syndrome population. Lastly, it will be important that our community is ready and willing to participate in Alzheimer’s trials as they become available.

Other key considerations

How are Kisunla and Leqembi different from each other?

Although the drugs in this class of amyloid lowering treatments are all antibodies, are administered via intravenous infusion, and work similarly; each has unique properties that affect amyloid plaque targeting, potency, and mechanism of action. In addition, the FDA-approved anti-amyloid drugs have different dosing schedules, efficacy, and side effect profiles. None of the drugs have been tested for safety among people with Down syndrome.

Should I advocate for my loved one to be prescribed an anti-amyloid therapy?

As with any medical and therapeutic treatments, the ultimate decision to take a drug should be made by patients, their families, and their trusted medical team. Due to the potential safety considerations outlined above, LuMind IDSC does not recommend people with Down syndrome take Kisunla or Leqembi at this time, except in the case of a clinical trial. This guidance aligns with the recommendations of the LuMind IDSC/NTG expert panel of medical doctors and clinicians, and with recently published appropriate use criteria for this class of drugs.

What can I do today to influence the future of my loved one with Down syndrome?

Families will play an important role in accelerating research into DS-AD. Increased participation is critical for the next phase of Down syndrome research. Please consider: