Firdapse (amifampridine) for LEMS
Firdapse® (currently approved in the EU) is the first and only approved drug for the symptomatic treatment of Lambert-Eaton Myasthenic Syndrome in adults, a rare autoimmune disease with the primary symptoms of muscle weakness.
Firdapse at a Glance
•Approved in the European Union
•Manufactured and commercialized by BioMarin in the European Union
•Designated as an orphan drug in the United States, the European Union and and Switzerland
Regulatory Status
In December 2009, Firdapse received marketing approval from the European Commission making it the first approved therapy for the symptomatic treatment of LEMS. Firdapse has been designated an orphan drug in the EU, which grants market exclusivity for ten years.
Commercialization Plan
Firdapse is commercialized by BioMarin in the European Union.
BioMarin is planning to conduct a Phase III Program and pursue FDA approval in the U.S.

欧盟批准兰伯特-伊顿肌无力综合症新药Amifampridine上市
欧盟委员会近日已批准BioMarin公司兰伯特-伊顿肌无力综合症（LEMS）治疗药Amifampridine上市销售。LEMS是一种罕见的自身免疫性疾病，最主要的症状为肌肉无力。在欧洲地区，每100万人当中大概有4-10人患有LEMS，在美国则有1200-3100人深受该病困扰。在这些患者当中，50%的人患有小细胞肺癌。
去年10月份，BioMarin通过收购Huxley制药公司而获得了该药的许可权。该公司负责人吉恩·雅克表示，Amifampridine是迄今为止首个获准的LEMS治疗药，它在欧洲将可以享受10年的独家销售权，它在美国也已被指定为罕见病治疗药。
他还透露，这种新药将在今年3月中旬进入欧洲市场，同时BioMarin相关人员将会尽快与FDA会面，对药物进入美国市场相关事宜进行磋商。除了治疗LEMS，该药治疗其他病症（特别是多发性硬化症）的研究也即将展开。
Amifampridine已是BioMarin公司获准进入市场的第四种产品，其他三种产品分别包括：治疗VI型黏多糖贮积症的Naglazyme（galsulfase）；治疗I型黏多糖贮积症的Aldurazyme （laronidase）；治疗苯丙酮酸尿症的Kuvan（sapropterin）。