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miglustat
Miglustat (OGT 918, ''N''-butyl-deoxynojirimycin) is a drug developed by Oxford GlycoSciences and marketed by Actelion and is used primarily to treat type I Gaucher disease (GD1). It is marketed under the trade name Zavesca.
==Medical uses==
Miglustat is used to treat adults with mild-to-moderate type I Gaucher disease for whom enzyme replacement therapy is unsuitable. It was approved in Europe in 2002〔European Medicines Agency. Human Medicines Database. (Zavesca (miglustat) ) Page Accessed 1 September 2014.〕 and by the US FDA in 2003.〔Actelion Press Release August 2003. (Zavesca approved -- first oral treatment option for type 1 Gaucher disease )〕
Miglustat is the first treatment to be approved for treating progressive neurological complications in people with Niemann–Pick disease, type C (NPC); it has been approved in Europe in 2009, Canada in 2010, and Japan in 2012, but not in the US where the FDA declined to approve it in 2010 and called for more data.〔UK Medicines Information. (New Drugs Online Report for miglustat )〕〔Staff, The Pharma Letter. 4 April 2012. (Actelion drops setipiprant, gets miglustat approval in Japan )〕〔Kevin Grogan for PharmaTimes. 10 March 2010. (FDA rejects Actelion's Zavesca for rare NP-C disease )〕〔Actelion Press Release. 23 March 2010 ( Zavesca® (Miglustat) First Treatment Available in Canada for Rare Progressive Niemann-Pick Type C Disease )〕
抄文引用元・出典: フリー百科事典『 ウィキペディア（Wikipedia）』
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