The Japanese pharmaceutical company Eisai Co., Ltd. has submitted a new drug application (NDA) in Japan for approval of ultra-high dose mecobalamin for treatment of ALS. Mecobalamin, one of the coenzyme forms of the vitamin B12, is already approved for treatment of other indications, such as peripheral neuropathies. In 2004, Eisai initiated a double-blind, placebo-controlled Phase II/III clinical trial of ultra-high dose mecobalamin in ALS, using time to event (ventilation or death) and change in the Japanese ALSFRS-R as primary endpoints. The results of the study suggested a trend toward a beneficial effect, but the difference in the primary endpoints between treated and untreated patients was not statistically significant. However, based on the pronounced treatment effect in two patient subgroups and the dire unmet need for therapies in ALS, Eisai has decided to move forward with the NDA submission in Japan. The results of the late stage clinical trial were presented at the 67th Annual American Academy of Neurology meeting in Washington, DC in April.
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