Hodgkinson L, Sorbera L, Graul A I
Thomson Reuters, London, UK.
Thomson Reuters, Barcelona, Spain.
Drugs Today (Barc). 2016 Mar;52(3):199-202. doi: 10.1358/dot.2016.52.3.2473590.
Highly anticipated as new disease-modifying treatments for Duchenne muscular dystrophy (DMD), therapeutics by BioMarin Pharmaceutical (Kyndrisa™; drisapersen) and Sarepta Therapeutics (eteplirsen; AVI-4658) both recently received negative FDA reviews and are now facing battles for approval in the U.S. At present, BioMarin is committed to working with the FDA to forge a pathway to approval following the failure of its NDA, while Sarepta awaits the formal decision on its NDA, which is expected by late May 2016. Despite the critical nature of both reviews, analysts consider that there is still a narrow possibility of approval of both drugs. According to Consensus forecasts from Thomson Reuters Cortellis for Competitive Intelligence, Kyndrisa is forecast to achieve sales of USD 533.71 million in 2021.
作为杜氏肌营养不良症（DMD）新的疾病修饰疗法备受期待的BioMarin制药公司的疗法（Kyndrisa™；drisapersen）和Sarepta Therapeutics公司的疗法（eteplirsen；AVI - 4658）最近均收到了美国食品药品监督管理局（FDA）的负面审评意见，目前正面临在美国获批的攻坚战。目前，BioMarin在其新药申请（NDA）失败后致力于与FDA合作，以寻求获批途径，而Sarepta则在等待其NDA的正式决定，预计该决定将于2016年5月底做出。尽管两份审评意见都很关键，但分析师认为这两种药物仍有微弱的获批可能性。根据汤森路透Cortellis竞争情报的共识预测，预计Kyndrisa在2021年的销售额将达到5.3371亿美元。
