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Monday, October 20, 2014
My relative in Poland has been diagniosed with Myasthemia Gravis. They heard about new drug Monarsen and would like to knoew if it is already available for purchase in USA, Canada or Europe.
Monarsen is a drug that works at the level of acetylcholine. Acquired myasthenia gravis is a disease where the neuromuscular junction is under attack at the acetylcholine receptor, causing disconnect between the nerve and the muscle, and then muscle weakness. Pyridostigmine (Mestinon) works by keeping what acetylcholine is present at the junction there so it can work longer by inhibiting acetylcholine esterase (AChE).
Monarsen (EN101) is a protein that causes a change the human AChE gene so that it is less effective, thus increasing acetylcholine?s duration at the neuromuscular junction. It is reported to have less cholinergic side effects than pyridostigmine. It is also better as far as timing: it only has to be taken once per day, where Mestinon has to be taken every 4-6 hours. Monarsen is still being investigated. Clinical comparison between monarsen and mestinon would be the best way to determine efficacy. It does have orphan drug status granted by the FDA from 2003 but still does not carry an FDA approved indication yet for myasthenia. I have heard about it off and on for several years, and I do wonder why it has not come to full indication yet. There are no trials listed on ClinicalTrials.gov currently. The drug is not available for prescription in the US at this time. Looking at the EMEA website, it did not appear that it has been approved by the European agency yet. I guess we will just have to wait and see.
Robert W Neel, IV, MD
Assistant Professor of Neurology
College of Medicine
University of Cincinnati