The Japanese pharmaceutical company Mitsubishi Tanabe Pharma Corporation has announced the submission of a New Drug Application (NDA) to the US FDA for edaravone for the treatment of ALS. The drug, also known as MCI-186, was approved for ALS in Japan in 2015 (see Jun 2015 news), and the same year was granted Orphan Drug Designation by the EMA and FDA in its oral formulation called TW001, which is under development by the Dutch biotechnology company, Treeway (see Mar 2015 news). Results of two Phase III clinical trials of the free radical scavenger previously conducted in Japan (see Jan 2016 news) suggested that a subgroup of ALS patients within 2 years of diagnosis and with >80% forced vital capacity benefit from the drug. It is still unclear whether the FDA will approve the drug based on these data, or will request Phase III studies in the US.
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