AMO Pharma announced that its investigational therapy for congenital myotonic dystrophy, AMO-02 (tideglusib), has been granted Fast Track designation by the Food and Drug Administration (FDA).

Myotonic dystrophy is the most common form of muscular dystrophy. Tideglusib is specifically intended for a severe form of myotonic dystrophy known as congenital DM1. 

Tideglusib is an inhibitor of glycogen synthase kinase 3 beta (GSK3ß), which is increased in patients with congenital myotonic dystrophy and Duchenne muscular dystrophy. Some animal models have demonstrated that GSK3ß inhibition may correct the activity of RNA binding proteins.  

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In preclinical studies, AMO-02 has shown efficacy in transgenic models and reversal of muscle cell differentiation deficits in ex vivo tissue samples. A Phase 2 clinical trial is currently being conducted for tideglusib. There is presently no treatment available for congenital DM1. The FDA’s Fast Track status is intended to expedite the review of treatments that have unmet medical needs.

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