Background

Amyotrophic lateral sclerosis (ALS) is a progressive and fatal neuromuscular disease with no curative therapies. Edaravone (Radicava^®) (Mitsubishi Tanabe Pharma Corporation, Tokyo, Japan), approved in the United States (US) for ALS in adults in 2017, was shown in a clinical trial to slow the rate of physical functional decline in ALS and is administered intravenously. The aim of this paper is to summarize the observed safety profile from real-world patient use during the first 3 years of edaravone availability in the US.

Methods

Edaravone usage data were collected, and adverse events (AEs) were identified from a postmarketing safety database from August 8, 2017 through August 7, 2020 (cutoff date).

Results

As of October 3, 2020, 5207 ALS patients had been treated with edaravone. As of August 7, 2020, the most commonly reported AEs included death (not specified), drug ineffective, disease progression, therapeutic response unexpected, fall, asthenia, fatigue, muscular weakness, gait disturbance, and dyspnea. The most commonly reported serious AEs (SAEs) included death (not specified), pneumonia, disease progression, ALS, fall, dyspnea, respiratory failure, device-related infection, hospitalization, and injection-site infection. There were 687 deaths, with 494 reported as death without specifying the cause. Deaths were most commonly attributed to ALS, disease progression, respiratory failure, or pneumonia. Review for administration-site reactions revealed 95 AEs, including 34 site infections, with 22 SAEs (all non-fatal). Five non-fatal SAEs of anaphylaxis were reported.

Conclusion

In the postmarketing reporting to date, no new safety signals were identified beyond those already known from the edaravone clinical trial program.

中文翻译：

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依达拉奉（Radicava®）上市后第三年美国安全数据分析

背景

肌萎缩侧索硬化症 (ALS) 是一种进行性和致命的神经肌肉疾病，没有治愈性疗法。依达拉奉 (Radicava ^® ) (Mitsubishi Tanabe Pharma Corporation, Tokyo, Japan) 于 2017 年在美国 (US) 批准用于成人 ALS，在一项临床试验中显示可减缓 ALS 身体机能衰退的速度，并给予静脉注射。本文的目的是总结在美国依达拉奉上市的前 3 年中，从真实世界患者使用中观察到的安全性概况。

方法

收集了依达拉奉的使用数据，并从 2017 年 8 月 8 日至 2020 年 8 月 7 日（截止日期）的上市后安全数据库中确定了不良事件 (AE)。

结果

截至 2020 年 10 月 3 日，已有 5207 名 ALS 患者接受了依达拉奉治疗。截至 2020 年 8 月 7 日，最常报告的 AE 包括死亡（未指定）、药物无效、疾病进展、意外的治疗反应、跌倒、虚弱、疲劳、肌肉无力、步态障碍和呼吸困难。最常报告的严重 AE (SAE) 包括死亡（未指定）、肺炎、疾病进展、ALS、跌倒、呼吸困难、呼吸衰竭、器械相关感染、住院和注射部位感染。有 687 人死亡，其中 494 人报告死亡，但未说明原因。死亡最常见的原因是 ALS、疾病进展、呼吸衰竭或肺炎。对给药部位反应的审查显示 95 例 AE，包括 34 例部位感染，其中 22 例 SAE（均非致命）。

结论

在迄今为止的上市后报告中，除了依达拉奉临床试验项目已知的安全信号外，没有发现新的安全信号。