FDA grants fast-track status for AMO Pharma’s congenital myotonic dystrophy treatment


The US Food and Drug Administration (FDA) has granted fast-track designation for AMO Pharma’s investigational therapy in development AMO-02 (tideglusib) to be used for the treatment of congenital myotonic dystrophy.

AMO Pharma is a biopharmaceutical company that focuses on debilitating diseases with limited or no treatment options.

Myotonic dystrophy is a progressive disease that affects approximately one in every 10,000 people. It is currently the most common form of muscular dystrophy.

The disease accompanies predominant symptoms, such as muscle weakness, intellectual / developmental impairment or cognitive / behavioural changes and is determined by several factors, including onset age.

There are currently no approved therapies available for the treatment of patients suffering from myotonic dystrophy.

"Congenital myotonic dystrophy is a devastating neuromuscular disease where affected patients currently have no treatment options available."

AMO Pharma chief executive officer Michael Snape said: “Congenital myotonic dystrophy is a devastating neuromuscular disease where affected patients currently have no treatment options available.

“The designation of fast-track status for our development programme for AMO-02 highlights both the urgent need for a treatment for patients and the potential for this novel therapy to offer a major advance in their care in the years ahead.”

AMO-02 is currently in clinical stage development and will be used to treat a severe form of myotonic dystrophy known as congenital DM1.

Glycogen synthase kinase 3 beta (GSK3ß) activity has been shown to have increased in cellular and animal models of congenital DM1 and Duchenne muscular dystrophy, as well as in muscle biopsies from patients.

Snape further added: “Our ongoing Phase II clinical trial for AMO-02 in the UK is the first sponsor-led clinical study in the treatment of congenital myotonic dystrophy and represents a historic milestone in research and a new era of hope for patients and their families affected by this serious condition.”