Pattee Gary L, Genge Angela, Couratier Philippe, Lunetta Christian, Sobue Gen, Aoki Masashi, Yoshino Hiide, Jackson Carlayne E, Wymer James, Salah Alejandro, Nelson Sally
Neurological Sciences Department, University of Nebraska Medical Center/Neurology Associates, Lincoln, Nebraska, USA.
Clinical Research and ALS Clinic, Montreal Neurological Institute and Hospital, Montreal, Canada.
Expert Rev Neurother. 2023 Jul-Dec;23(10):859-866. doi: 10.1080/14737175.2023.2251687. Epub 2023 Sep 12.
Amyotrophic lateral sclerosis (ALS) is a progressive and incurable neurodegenerative disease. While pharmacotherapy options remain limited, the Food and Drug Administration (FDA) approved intravenous (IV) and oral edaravone for the treatment of ALS in 2017 and 2022, respectively. With the addition of oral edaravone, patients with ALS may exclusively use oral medications.
The authors performed a review of the published literature using the United States (US) National Library of Medicine's PubMed.gov resource to describe the pharmacokinetics, pharmacodynamics, safety, and efficacy of oral edaravone, as well as pertinent completed and ongoing clinical trials, including the oral edaravone clinical trial development program. The clinical profile of oral edaravone is also discussed.
Edaravone has been shown to slow the rate of motor function deterioration experienced by patients with ALS. As the oral formulation has been approved, patients with ALS may use it alone or in combination with other approved therapeutics. Additional clinical trials and real-world evidence are ongoing to gain further understanding of the clinical profile of oral edaravone.
肌萎缩侧索硬化症(ALS)是一种进行性且无法治愈的神经退行性疾病。虽然药物治疗选择仍然有限,但美国食品药品监督管理局(FDA)分别于2017年和2022年批准了静脉注射(IV)和口服依达拉奉用于治疗ALS。随着口服依达拉奉的加入,ALS患者可以仅使用口服药物。
作者使用美国国立医学图书馆的PubMed.gov资源对已发表的文献进行了综述,以描述口服依达拉奉的药代动力学、药效学、安全性和有效性,以及相关的已完成和正在进行的临床试验,包括口服依达拉奉临床试验开发项目。还讨论了口服依达拉奉的临床概况。
依达拉奉已被证明可减缓ALS患者运动功能恶化的速度。由于口服制剂已获批准,ALS患者可以单独使用它或与其他已批准的治疗方法联合使用。正在进行更多的临床试验和真实世界证据收集,以进一步了解口服依达拉奉的临床概况。