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3,4-Diaminopyridine (or 3,4-DAP) is an organic compound with the formula C5H3N(NH2)2. It is formally derived from pyridine by substitution of the 3 and 4 positions with an amino group. The compound 3,4-diaminopyridine has the International Nonproprietary Name amifampridine and is used as a drug, predominantly in the treatment of a number of rare muscle diseases. The phosphate salt of amifampridine is a more stable formulation that does not require refrigeration; it is marketed in the EU by BioMarin Pharmaceutical under the trade name Firdapse and designated as an orphan drug in the EU.〔(【引用サイトリンク】 url = http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/medicines/001032/human_med_001298.jsp )〕 In the United States, amifampridine is under investigation for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) by Catalyst Pharmaceuticals, and was granted a breakthrough therapy designation by the FDA in 2013.〔(【引用サイトリンク】 Rare Disease: Catalyst Pharmaceutical Receives 20th FDA Breakthrough Therapy Designation )〕 In a Phase 3 clinical trial, it demonstrated superiority over placebo in both co-primary endpoints.〔(American Neurological Association 2013 Meeting Poster ID: S737WIP )〕〔(Muscular Dystrophy Association Press Release )〕 LEMS patients can receive amifampridine at no cost under an ongoing expanded access program. A study to develop patient entry criteria for a clinical trial of the free base form of amifampridine has also been completed by Jacobus Pharmaceutical Company. This form remains available at no cost to patients with Lambert-Eaton myasthenic syndrome and congenital myasthenic syndromes under a long-standing compassionate use program, with FDA oversight, from Jacobus Pharmaceutical Company (see also Medical uses and Economics, below). == Medical uses == A 2011 systematic review from the Cochrane Collaboration found data favoring its use in LEMS. Although not approved for pharmaceutical use in the United States, amifampridine is available under compassionate use regulations for the treatment of LEMS. Amifampridine is also used to treat many of the congenital myasthenic syndromes, particularly those with defects in choline acetyltransferase, downstream kinase 7, and those where any kind of defect causes "fast channel" behaviour of the acetylcholine receptor. In the US, amifampridine is under development as an orphan drug for congenital myasthenic syndrome〔()〕 and 3,4-diaminopyridine is available at no cost for patients with congenital myasthenic syndromes under a long-standing compassionate use program from Jacobus Pharmaceutical Co. Amifampridine has also been proposed for the treatment of multiple sclerosis, but a 2002 systematic review found that there was little unbiased data to support its use in MS. 抄文引用元・出典: フリー百科事典『 ウィキペディア（Wikipedia）』 ■ウィキペディアで「3,4-Diaminopyridine」の詳細全文を読む スポンサード リンク
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