Firdapse first for symptomatic LEMS treatment

BioMarin Europe is introducing the first authorised therapy for the symptomatic treatment of Lambert-Eaton Myasthenic Syndrome (LEMS) in adults.

Firdapse tablets contain 18.98mg of amifampridine phosphate (equivalent to 10mg amifampridine).

LEMS is a rare antibody-mediated autoimmune disease of neuromuscular transmission. Amifampridine is recommended as first-line treatment for LEMS by the European Federation of Neurological Sciences task force, but has previously only been available as an unlicensed product or compounded amifampridine base.

Treatment should be initiated by a doctor experienced in the treatment of LEMS. The tablets should be given in divided doses, three or four times a day. The recommended starting dose is 15mg a day, which can be increased in 5mg increments every four to five days to a maximum of 60mg per day. No single dose should exceed 20mg

欧盟委员会近日批准Bio Marin公司的兰伯特-伊顿肌无力综合征（LEMS）治疗药Amifampridine上市销售。LEMS是一种罕见的自身免疫性疾病，其最主要的症状为肌无力。
    在欧洲地区，每100万人当中约有4～10人患有LEMS。在这些患者当中，50%的人患有小细胞肺癌。Bio Marin公司负责人表示，Amifampridine在欧洲将可以享受10年的独家销售权，在美国该药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，Bio Marin公司相关人员将会尽快与美国食品药品管理局（FDA）接洽，对该药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。