Biopharmaceutical company AMO Pharma Limited revealed on Tuesday the receipt of the US Food and Drug Administration (FDA) Fast Track designation for AMO-02 to treat congenital myotonic dystrophy.

Myotonic dystrophy is reportedly the most common form of muscular dystrophy, affecting one in 10,000 people. It is a progressive disease and the predominance of symptoms such as muscle weakness, intellectual/developmental impairment or cognitive/behavioural changes is determined by a number of factors, including age at onset.

According to the company, AMO-02 (tideglusib) is in clinical stage development for the treatment of the severe form of myotonic dystrophy known as congenital DM1.

In the company's cellular and animal models of congenital DM1 and Duchenne muscular dystrophy, as well as in muscle biopsies from patients, the activity of glycogen synthase kinase 3 beta (GSK3ß) has been shown to be increased. Inhibitors of GSK3ß have been shown to correct the activity of RNA binding proteins, such as CUGBP1.

The company said AMO-02 is an inhibitor of GSK3ß that has demonstrated pre-clinical efficacy in transgenic models and reversal of muscle cell differentiation deficits in ex vivo tissue samples derived from patients with congenital DM1.