Eli Lilly's Kisunla (donanemab-azbt), an amyloid plaque-targeting Alzheimer's disease (AD) drug, has been approved by the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK. However, the National Institute for Health and Care Excellence (NICE) has issued draft guidance not recommending its use on the National Health Service (NHS) due to cost-effectiveness concerns.
Regulatory Approval and Clinical Evidence
Kisunla is approved for treating mild cognitive impairment and mild dementia due to AD in adults who have one or no copies of the apolipoprotein E4 gene. The drug is administered as an intravenous infusion every four weeks. The MHRA's approval was supported by positive results from the late-stage TRAILBLAZER-ALZ 2 study, which demonstrated statistically significant less clinical progression in patients treated with Kisunla compared to those receiving a placebo.
According to Lilly, Kisunla is the "first and only" amyloid plaque-targeting therapy with evidence to support stopping treatment when amyloid plaques are removed. This offers a potential advantage over other similar treatments that require continuous administration.
NICE's Assessment and Cost Concerns
Despite the regulatory approval, NICE has stated in draft guidance that the cost of providing Kisunla "cannot currently be considered good value for the taxpayer." This means that eligible patients who wish to receive the drug may have to pay for it privately. NICE's independent committee noted that the clinical benefit was "relatively small" and that there are "significant uncertainties" about the extent and duration of the benefit after stopping treatment. The committee also raised concerns about "significant health risks" associated with the treatment, based on clinical trial evidence.
NICE has requested additional information from Lilly and NHS England to address the uncertainties in the evidence and is expected to make a final decision in 2025. This decision follows a similar rejection of Biogen and Eisai's AD treatment, Leqembi (lecanemab), which NICE deemed to have benefits that were "too small to justify the costs."
Patient Advocacy and Future Prospects
Fiona Carragher, chief policy and research officer at Alzheimer's Society, described NICE's decision as "disheartening." However, she noted that there are approximately 20 AD drugs in late-stage clinical trials, suggesting that "more drugs will be submitted for approval within the next few years." This indicates a promising future for AD treatment options, with potential for more effective, safer, and cheaper therapies.
Peter Almond, a patient treated in clinical trials of Kisunla, expressed his disappointment, stating, "Today's news means me and many others won't benefit from this drug. Without donanemab, I have no idea what the future holds. The thought of my disease quickly progressing, and not knowing where or who I am, terrifies me."
Alzheimer's Research UK reported that NICE's cost-effectiveness estimate for donanemab is five to six times above what it normally considers an acceptable use of NHS resources, highlighting the significant divide between the agency and the company regarding affordability.
Impact and Context
An estimated 982,000 people are living with dementia in the UK, with AD accounting for up to 75% of cases. The neurodegenerative disease slowly destroys memory and thinking skills, eventually impairing the ability to perform simple tasks. Lilly estimates the total cost of dementia to the UK at £42 billion in 2024. The current situation underscores the challenges in balancing the potential benefits of new Alzheimer's treatments with the economic realities of healthcare provision.
