✅ **新药获批与适用范围界定：** 礼来公司的多那单抗（donanemab）已获得欧洲委员会批准，用于治疗早期症状性阿尔茨海默病。此次批准是在欧洲药品管理局（EMA）最初因安全顾虑而未予推荐后，经过进一步评估得出的。新批准的适应症限定为轻度认知障碍和轻度痴呆阶段的患者，并且排除了携带两种或一种ApoE4基因拷贝的个体，这表明了药物在特定人群中的疗效和安全性考量。

💡 **关键疗效与作用机制：** 多那单抗是一种每月一次的静脉注射疗法，其核心优势在于它是首个被证明能在清除大脑中的淀粉样蛋白斑块至极低水平后，帮助患者完成治疗过程的药物。这一机制基于III期TRAILBLAZER-ALZ 2临床试验的结果，该试验显示多那单抗能够显著延缓早期阿尔茨海默病患者的认知和功能衰退，并且越早诊断和治疗，患者的反应越好。

📈 **长期疗效与市场前景：** TRAILBLAZER-ALZ 2试验的长期数据显示，接受早期治疗的患者在三年内疾病进展到下一阶段的风险降低了27%，证明了药物的持续益处。尽管分析师预测其在主要市场具有可观的销售潜力，但其定价策略和在英国国家医疗服务体系（NHS）的接受度仍是潜在的挑战，此前英国的成本效益监管机构NICE已拒绝支持其在NHS上的使用。

Eli Lilly’s Kisunla (donanemab) has been approved by the European Commission (EC) to treat early symptomatic Alzheimer’s disease, overcoming its knockback earlier this year.

In March European Medicines Agency (EMA) advisors refused to back its approval, believing its benefits were not enough to outweigh the risk of potentially fatal events due to amyloid-related imaging abnormalities (ARIA), involving swelling and potential bleeding in the brain.

After re-examining the drug the EMA’s committee for Medicinal Products for Human Use (CHMP) recommended Kisunla for approval, but only for a subset of the patient population.

Consequently, the monoclonal antibody is now indicated for adults with mild cognitive impairment and individuals with mild dementia stages of Alzheimer’s, but only for those that do not have a copy of the ApoE4 gene, a certain form of the gene for the protein apolipoprotein E, or people who have only one copy of the gene.

Clinical efficacy

Kisunla, which is administered as a monthly infusion, is the first amyloid plaque-targeting therapy to show evidence of helping patients to complete their treatment course when amyloid plaques are reduced to minimal levels, according to Lilly.

Its new European authorisation is based on the phase III TRAILBLAZER-ALZ 6 and TRAILBLAZER-ALZ 2 clinical trials.

Patrik Jonsson, Executive Vice President and President of Lilly International, explained that in the TRAILBLAZER-ALZ 2 trial, “Kisunla demonstrated meaningful results in people with early symptomatic Alzheimer’s disease by significantly slowing cognitive and functional decline… [and] the earlier patients are identified, diagnosed, and treated with Kisunla, the greater the response to treatment”.

Long-term clinical data from an extension study of the TRAILBLAZER-ALZ 2 trial released in July showed that the drug provided sustained benefit in early Alzheimer’s over a three-year period. Specifically, participants treated earlier gained a 27 percent reduction in their risk of progression to the next stage of disease.

Analysts at GlobalData forecasted last year that Kisunla could produce sales of approximately $2 billion in 2030 in the US, France, Germany, Italy, Spain, UK, Japan, and China.

However, its pricing could be an issue and the UK’s cost and clinical effectiveness watchdog National Institute for Health and Care Excellence (NICE) earlier this year declined to back its use on the NHS.

The post Lilly wins European Alzheimer’s approval for Kisunla after regulatory setbacks appeared first on European Pharmaceutical Review.